Healthy Volunteers, Hemophilia A
Conditions
Brief summary
This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects.
Interventions
DRUGEmicizumab
Participants will receive a single 1 milligram per kilogram of body weight (mg/kg) subcutaneous dose of emicizumab on Day 1.
Sponsors
Hoffmann-La Roche
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
20 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
* Healthy Chinese male subjects, aged 20-45 years inclusive at the time of screening * Chinese subjects must have Chinese parents and grandparents, all of whom were born in China * A body mass index (BMI) between 19 and 24 kilograms per height in meters squared (kg/m\^2), inclusive * Able to participate and willing to give written informed consent and to comply with the study requirements
Exclusion criteria
* Any history or presence of a clinically significant disorder, or any other condition or disease which in the judgement of the investigator would place the subject at undue risk; interfere with absorption, distribution, metabolism, and excretion of emicizumab; or interfere with the ability of the subject to complete the study * Major illness within 1 month prior to dosing, and/or any condition which could relapse during or immediately after the study * Use of any prescribed or over-the-counter (OTC) medication or herbal medicine taken within 14 days prior to dosing or within 5 times the elimination half-life of the medication prior to dosing (whichever is longer), with some exceptions * Any significant donation/loss of blood or plasma (greater than 450 milliliters) within the 3 months prior to dosing * Regular smoker with consumption of more than 10 cigarettes per day or the equivalent amount of tobacco * Participation within a clinical study with an investigational drug or device within the last 3 months prior to dosing * Any clinically relevant history of hypersensitivity or allergic reactions, either spontaneous or following drug administration or exposure to foods or environmental agents * Previous or concomitant thromboembolic disease such as deep vein thrombosis (DVT) or signs of thromboembolic disease, or family history of thromboembolic disorder such as serious DVT * At high risk for thrombotic microangiopathy (e.g., have a previous medical or family history of thrombotic microangiopathy), in the investigator's judgment * Previous or concomitant autoimmune or connective tissue disease * History of tuberculosis or active tuberculosis with positive test result at screening * Any other reason that, in the judgment of the investigator, would render the subject unsuitable for study participation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Maximum Observed Plasma Concentration (Cmax) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Time to Cmax (Tmax) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Apparent Terminal Half-Life (t1/2) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Apparent Clearance (CL/F) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Apparent Volume of Distribution (Vz/F) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Mean Residence Time (MRT) of Emicizumab | Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods. |
| Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | From screening to study completion (20 weeks) | The WHO Toxicity Grading Scale was used for assessing adverse event severity. Any adverse event not specifically listed in the WHO Toxicity Grading Scale was assessed according to the following levels of severity: Grade 1 is mild; Grade 2 is moderate; Grade 3 is severe; and Grade 4 is life-threatening. Investigator text for adverse events were encoded using the Medical Dictionary for Regulatory Activities (MedDRA) version 21.1. After informed consent had been obtained but prior to initiation of study drug, only serious adverse events (SAEs) caused by a protocol-mandated intervention were to have been reported. After initiation of study drug, all adverse events, regardless of relationship to study drug, were to have been reported until the participant completed his last study visit. After this period, any SAEs believed to be related to prior study drug treatment were to be reported. |
| Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Predose at Baseline (Day 1) and postdose on Days 57 and 113 | Participants were considered to be 'ADA Negative (Treatment Unaffected)' if baseline and all post-baseline samples were negative, or if they were ADA positive at baseline but did not have any post-baseline samples with a titer that was at least 4-fold greater than the titer of the baseline sample. 'Total ADA Positive' is the sum of all participants who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Induced)', those who were ADA negative at baseline and tested positive for ADA following study drug administration; and 'ADA Positive (Treatment Boosted)', those who were pre-dose ADA positive and had post-baseline samples with a titer that was at least 4-fold greater compared to the baseline measurement. |
| Number of Participants With Laboratory Test Abnormalities | Baseline and Days 2, 4, 8, 11, 15, 29, 43, 57, 71, 85, and 113 | The number of participants with a laboratory abnormality during treatment (numerator) is reported among the 'number analyzed' in the table below (denominator), which represents the number of participants without that abnormality at baseline (last observation prior to initiation of study drug). Note that samples from all participants were analyzed for each laboratory parameter. Values falling above or below the Roche predefined standard reference range were laboratory abnormalities labelled accordingly as 'high' or 'low'. Not every laboratory abnormality qualified as an adverse event; only if it was accompanied by clinical symptoms, resulted in a change in study treatment or in a medical intervention, or was clinically significant in the investigator's judgment. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Baseline and Days 2, 11, 29, 57, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Baseline and Days 2, 11, 29, 57 and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Baseline and Days 2, 11, 29, 57 and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Baseline and Days 2, 11, 29, 57 and 113 | The INR is a standardized measure of the prothrombin time. Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Erythrocyte mean corpuscular hemoglobin (MCH) is a measure of the average amount of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Erythrocyte mean corpuscular volume is a measure of the average volume of a red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Erythrocyte mean corpuscular hemoglobin concentration (MCHC) is a measure of the average concentration of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113 | Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Number of Participants by Test Results for Blood in Urine by Timepoint | Baseline and Days 2, 8, 29, 57, 85, and 113 | Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for blood in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Number of Participants by Test Results for Glucose in Urine by Timepoint | Baseline and Days 2, 8, 29, 57, 85, and 113 | Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for glucose in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Number of Participants by Test Results for Protein in Urine by Timepoint | Baseline and Days 2, 8, 29, 57, 85, and 113 | Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for protein in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug. |
| Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113 | Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in ECG Results by Timepoint: PR Duration | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in ECG Results by Timepoint: QRS Duration | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in ECG Results by Timepoint: QT Duration | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Change From Baseline in ECG Results by Timepoint: RR Duration | Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113 | The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug. |
| Number of Participants With Concomitant Medications | From screening to study completion (20 weeks) | The original terms recorded by the investigator for concomitant medications were standardized by the sponsor by assigning preferred terms. The duration of treatment with the concomitant medications ranged from 1 day to 5 days. Except for 1 participant who was treated during the in-clinic period (Days -1 to 4), all other concomitant medications were recorded during the ambulatory period (Days 6 to 113). |
Countries
China
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Emicizumab Participants received a single 1 milligram per kilogram of body weight (mg/kg) subcutaneous dose of emicizumab under fasting conditions on Day 1. | 16 |
| Total | 16 |
Baseline characteristics
| Characteristic | Emicizumab |
|---|---|
| Age, Continuous | 31.6 years STANDARD_DEVIATION 6.7 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 16 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 16 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 16 |
| other Total, other adverse events | 14 / 16 |
| serious Total, serious adverse events | 0 / 16 |
Outcome results
Primary
Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab | 287 μg*day/mL | Standard Deviation 74.2 |
Primary
Maximum Observed Plasma Concentration (Cmax) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | Maximum Observed Plasma Concentration (Cmax) of Emicizumab | 7.11 microgram per milliliter (μg/mL) | Standard Deviation 1.77 |
Secondary
Apparent Clearance (CL/F) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | Apparent Clearance (CL/F) of Emicizumab | 235 milliliter per day (mL/day) | Standard Deviation 88 |
Secondary
Apparent Terminal Half-Life (t1/2) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | Apparent Terminal Half-Life (t1/2) of Emicizumab | 26.7 day | Standard Deviation 4.25 |
Secondary
Apparent Volume of Distribution (Vz/F) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | Apparent Volume of Distribution (Vz/F) of Emicizumab | 8870 mL | Standard Deviation 2950 |
Secondary
AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab | 268 μg*day/mL | Standard Deviation 63.7 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Baseline (BL) - Value at Visit | 45.43 gram per Liter (g/L) | Standard Deviation 1.834 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 2 | -0.43 gram per Liter (g/L) | Standard Deviation 2.391 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 4 | 1.03 gram per Liter (g/L) | Standard Deviation 2.016 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 8 | 0.29 gram per Liter (g/L) | Standard Deviation 2.095 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 15 | 0.08 gram per Liter (g/L) | Standard Deviation 1.928 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 29 | -0.18 gram per Liter (g/L) | Standard Deviation 1.602 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 43 | -0.41 gram per Liter (g/L) | Standard Deviation 1.242 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 57 | -1.24 gram per Liter (g/L) | Standard Deviation 2.298 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 71 | -0.58 gram per Liter (g/L) | Standard Deviation 1.983 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 85 | -1.56 gram per Liter (g/L) | Standard Deviation 2.308 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Change From BL at Day 113 | -0.26 gram per Liter (g/L) | Standard Deviation 1.732 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Post-BL Minimum Change From BL | -2.87 gram per Liter (g/L) | Standard Deviation 1.967 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration | Post-BL Maximum Change From BL | 1.98 gram per Liter (g/L) | Standard Deviation 1.521 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Baseline (BL) - Value at Visit | 85.38 unit per Liter (U/L) | Standard Deviation 19.5 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 2 | 0.50 unit per Liter (U/L) | Standard Deviation 6.792 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 4 | 2.50 unit per Liter (U/L) | Standard Deviation 6.542 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 8 | 1.06 unit per Liter (U/L) | Standard Deviation 5.813 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 15 | 2.38 unit per Liter (U/L) | Standard Deviation 9.15 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 29 | 1.44 unit per Liter (U/L) | Standard Deviation 8.959 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 43 | 0.56 unit per Liter (U/L) | Standard Deviation 8.756 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 57 | -3.69 unit per Liter (U/L) | Standard Deviation 9.918 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 71 | -0.94 unit per Liter (U/L) | Standard Deviation 11.198 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 85 | -4.50 unit per Liter (U/L) | Standard Deviation 7.589 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Change From BL at Day 113 | -0.38 unit per Liter (U/L) | Standard Deviation 11.684 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Post-BL Minimum Change From BL | -9.81 unit per Liter (U/L) | Standard Deviation 7.556 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration | Post-BL Maximum Change From BL | 9.94 unit per Liter (U/L) | Standard Deviation 7.398 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 43 | -0.25 micromole per Liter (μmol/L) | Standard Deviation 3.709 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Baseline (BL) - Value at Visit | 13.83 micromole per Liter (μmol/L) | Standard Deviation 7.556 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 2 | 0.82 micromole per Liter (μmol/L) | Standard Deviation 2.921 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 4 | -0.42 micromole per Liter (μmol/L) | Standard Deviation 4.47 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 8 | -1.10 micromole per Liter (μmol/L) | Standard Deviation 3.713 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 15 | -0.61 micromole per Liter (μmol/L) | Standard Deviation 5.909 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 29 | -0.56 micromole per Liter (μmol/L) | Standard Deviation 4.688 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 57 | -0.96 micromole per Liter (μmol/L) | Standard Deviation 4.034 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 71 | 1.67 micromole per Liter (μmol/L) | Standard Deviation 7.467 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 85 | 0.54 micromole per Liter (μmol/L) | Standard Deviation 3.861 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Change From BL at Day 113 | -0.12 micromole per Liter (μmol/L) | Standard Deviation 5.57 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Post-BL Minimum Change From BL | -3.94 micromole per Liter (μmol/L) | Standard Deviation 4.749 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration | Post-BL Maximum Change From BL | 5.54 micromole per Liter (μmol/L) | Standard Deviation 5.909 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 29 | 0.43 millimole per Liter (mmol/L) | Standard Deviation 1.071 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Baseline (BL) - Value at Visit | 4.46 millimole per Liter (mmol/L) | Standard Deviation 0.848 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 2 | -0.58 millimole per Liter (mmol/L) | Standard Deviation 0.877 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 4 | -0.73 millimole per Liter (mmol/L) | Standard Deviation 0.649 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 8 | 0.26 millimole per Liter (mmol/L) | Standard Deviation 1.074 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 15 | 0.43 millimole per Liter (mmol/L) | Standard Deviation 1.543 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 43 | 0.38 millimole per Liter (mmol/L) | Standard Deviation 0.959 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 57 | 0.39 millimole per Liter (mmol/L) | Standard Deviation 0.874 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 71 | 0.36 millimole per Liter (mmol/L) | Standard Deviation 0.958 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 85 | 0.16 millimole per Liter (mmol/L) | Standard Deviation 0.888 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Change From BL at Day 113 | 0.13 millimole per Liter (mmol/L) | Standard Deviation 0.573 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Post-BL Minimum Change From BL | -1.07 millimole per Liter (mmol/L) | Standard Deviation 0.587 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration | Post-BL Maximum Change From BL | 1.42 millimole per Liter (mmol/L) | Standard Deviation 0.97 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Baseline (BL) - Value at Visit | 104.69 millimole per Liter (mmol/L) | Standard Deviation 1.957 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 2 | -0.69 millimole per Liter (mmol/L) | Standard Deviation 2.549 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 4 | -0.63 millimole per Liter (mmol/L) | Standard Deviation 2.363 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 8 | 0.13 millimole per Liter (mmol/L) | Standard Deviation 2.579 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 15 | 0.06 millimole per Liter (mmol/L) | Standard Deviation 2.175 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 29 | -1.56 millimole per Liter (mmol/L) | Standard Deviation 2.279 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 43 | -0.06 millimole per Liter (mmol/L) | Standard Deviation 1.731 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 57 | 0.75 millimole per Liter (mmol/L) | Standard Deviation 2.769 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 71 | -0.50 millimole per Liter (mmol/L) | Standard Deviation 2.757 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 85 | 0.13 millimole per Liter (mmol/L) | Standard Deviation 2.63 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Change From BL at Day 113 | -0.13 millimole per Liter (mmol/L) | Standard Deviation 2.217 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Post-BL Minimum Change From BL | -2.94 millimole per Liter (mmol/L) | Standard Deviation 2.016 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration | Post-BL Maximum Change From BL | 2.31 millimole per Liter (mmol/L) | Standard Deviation 2.12 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 2 | 0.02 millimole per Liter (mmol/L) | Standard Deviation 0.393 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Baseline (BL) - Value at Visit | 3.88 millimole per Liter (mmol/L) | Standard Deviation 1.103 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 4 | 0.07 millimole per Liter (mmol/L) | Standard Deviation 0.439 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 8 | -0.03 millimole per Liter (mmol/L) | Standard Deviation 0.383 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 15 | -0.04 millimole per Liter (mmol/L) | Standard Deviation 0.451 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 29 | 0.03 millimole per Liter (mmol/L) | Standard Deviation 0.372 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 43 | -0.06 millimole per Liter (mmol/L) | Standard Deviation 0.441 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 57 | -0.04 millimole per Liter (mmol/L) | Standard Deviation 0.459 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 71 | -0.01 millimole per Liter (mmol/L) | Standard Deviation 0.466 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 85 | -0.13 millimole per Liter (mmol/L) | Standard Deviation 0.515 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Change From BL at Day 113 | 0.01 millimole per Liter (mmol/L) | Standard Deviation 0.394 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Post-BL Minimum Change From BL | -0.47 millimole per Liter (mmol/L) | Standard Deviation 0.468 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration | Post-BL Maximum Change From BL | 0.47 millimole per Liter (mmol/L) | Standard Deviation 0.222 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Baseline (BL) - Value at Visit | 0.50 milligram per Liter (mg/L) | Standard Deviation 0.568 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 15 | 0.75 milligram per Liter (mg/L) | Standard Deviation 1.197 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Post-BL Minimum Change From BL | -0.20 milligram per Liter (mg/L) | Standard Deviation 0.397 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 2 | 0.23 milligram per Liter (mg/L) | Standard Deviation 0.456 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 4 | 0.11 milligram per Liter (mg/L) | Standard Deviation 0.477 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 8 | 0.54 milligram per Liter (mg/L) | Standard Deviation 1.721 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 29 | 1.01 milligram per Liter (mg/L) | Standard Deviation 1.289 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 43 | 0.60 milligram per Liter (mg/L) | Standard Deviation 1.849 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 57 | 0.27 milligram per Liter (mg/L) | Standard Deviation 0.827 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 71 | 0.13 milligram per Liter (mg/L) | Standard Deviation 0.41 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 85 | 0.12 milligram per Liter (mg/L) | Standard Deviation 0.595 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Change From BL at Day 113 | 1.24 milligram per Liter (mg/L) | Standard Deviation 3.517 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration | Post-BL Maximum Change From BL | 3.14 milligram per Liter (mg/L) | Standard Deviation 3.641 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Baseline (BL) - Value at Visit | 121.63 unit per Liter (U/L) | Standard Deviation 81.915 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 2 | -41.13 unit per Liter (U/L) | Standard Deviation 54.76 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 4 | -25.81 unit per Liter (U/L) | Standard Deviation 107.494 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 8 | -22.19 unit per Liter (U/L) | Standard Deviation 54.034 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 15 | -0.94 unit per Liter (U/L) | Standard Deviation 39.834 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 29 | 8.50 unit per Liter (U/L) | Standard Deviation 122.645 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 43 | -21.00 unit per Liter (U/L) | Standard Deviation 53.551 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 57 | 8.00 unit per Liter (U/L) | Standard Deviation 152.217 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 71 | -19.63 unit per Liter (U/L) | Standard Deviation 51.798 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 85 | -20.00 unit per Liter (U/L) | Standard Deviation 77.283 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Change From BL at Day 113 | -18.81 unit per Liter (U/L) | Standard Deviation 71.191 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Post-BL Minimum Change From BL | -52.38 unit per Liter (U/L) | Standard Deviation 72.902 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration | Post-BL Maximum Change From BL | 92.50 unit per Liter (U/L) | Standard Deviation 155.002 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 85 | -1.50 micromole per Liter (μmol/L) | Standard Deviation 4.662 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Baseline (BL) - Value at Visit | 83.19 micromole per Liter (μmol/L) | Standard Deviation 7.195 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 2 | -0.56 micromole per Liter (μmol/L) | Standard Deviation 7.071 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 4 | -0.31 micromole per Liter (μmol/L) | Standard Deviation 5.147 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 8 | -1.25 micromole per Liter (μmol/L) | Standard Deviation 4.919 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 15 | -2.00 micromole per Liter (μmol/L) | Standard Deviation 9.452 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 29 | -3.69 micromole per Liter (μmol/L) | Standard Deviation 4.672 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 43 | -2.06 micromole per Liter (μmol/L) | Standard Deviation 5.434 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 57 | -2.38 micromole per Liter (μmol/L) | Standard Deviation 5.488 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 71 | -1.06 micromole per Liter (μmol/L) | Standard Deviation 7.344 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Change From BL at Day 113 | -0.19 micromole per Liter (μmol/L) | Standard Deviation 6.327 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Post-BL Minimum Change From BL | -9.25 micromole per Liter (μmol/L) | Standard Deviation 5.17 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration | Post-BL Maximum Change From BL | 6.13 micromole per Liter (μmol/L) | Standard Deviation 6.109 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 2 | 0.56 micromole per Liter (μmol/L) | Standard Deviation 0.395 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Post-BL Minimum Change From BL | -0.39 micromole per Liter (μmol/L) | Standard Deviation 0.396 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Baseline (BL) - Value at Visit | 1.63 micromole per Liter (μmol/L) | Standard Deviation 0.567 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 4 | 0.29 micromole per Liter (μmol/L) | Standard Deviation 0.452 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 8 | 0.00 micromole per Liter (μmol/L) | Standard Deviation 0.327 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 15 | -0.07 micromole per Liter (μmol/L) | Standard Deviation 0.521 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 29 | 0.12 micromole per Liter (μmol/L) | Standard Deviation 0.548 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 43 | 0.12 micromole per Liter (μmol/L) | Standard Deviation 0.308 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 57 | -0.03 micromole per Liter (μmol/L) | Standard Deviation 0.342 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 71 | 0.23 micromole per Liter (μmol/L) | Standard Deviation 0.509 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 85 | 0.17 micromole per Liter (μmol/L) | Standard Deviation 0.515 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Change From BL at Day 113 | 0.07 micromole per Liter (μmol/L) | Standard Deviation 0.48 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration | Post-BL Maximum Change From BL | 0.78 micromole per Liter (μmol/L) | Standard Deviation 0.448 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Baseline (BL) - Value at Visit | 5.03 millimole per Liter (mmol/L) | Standard Deviation 0.425 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 2 | -0.33 millimole per Liter (mmol/L) | Standard Deviation 0.384 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 4 | -0.22 millimole per Liter (mmol/L) | Standard Deviation 0.5 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 8 | -0.16 millimole per Liter (mmol/L) | Standard Deviation 0.441 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 15 | -0.27 millimole per Liter (mmol/L) | Standard Deviation 0.382 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 29 | -0.08 millimole per Liter (mmol/L) | Standard Deviation 0.364 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 43 | -0.08 millimole per Liter (mmol/L) | Standard Deviation 0.435 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 57 | -0.22 millimole per Liter (mmol/L) | Standard Deviation 0.515 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 71 | -0.20 millimole per Liter (mmol/L) | Standard Deviation 0.322 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 85 | -0.25 millimole per Liter (mmol/L) | Standard Deviation 0.346 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Change From BL at Day 113 | -0.21 millimole per Liter (mmol/L) | Standard Deviation 0.47 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Post-BL Minimum Change From BL | -0.64 millimole per Liter (mmol/L) | Standard Deviation 0.388 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration | Post-BL Maximum Change From BL | 0.24 millimole per Liter (mmol/L) | Standard Deviation 0.386 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 71 | 0.14 millimole per Liter (mmol/L) | Standard Deviation 0.314 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 85 | 0.28 millimole per Liter (mmol/L) | Standard Deviation 0.395 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 113 | 0.33 millimole per Liter (mmol/L) | Standard Deviation 0.429 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Post-BL Minimum Change From BL | -0.19 millimole per Liter (mmol/L) | Standard Deviation 0.318 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Post-BL Maximum Change From BL | 1.02 millimole per Liter (mmol/L) | Standard Deviation 0.49 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Baseline (BL) - Value at Visit | 0.98 millimole per Liter (mmol/L) | Standard Deviation 0.446 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 2 | 0.37 millimole per Liter (mmol/L) | Standard Deviation 0.377 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 4 | 0.57 millimole per Liter (mmol/L) | Standard Deviation 0.517 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 8 | 0.03 millimole per Liter (mmol/L) | Standard Deviation 0.35 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 15 | 0.22 millimole per Liter (mmol/L) | Standard Deviation 0.497 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 29 | 0.15 millimole per Liter (mmol/L) | Standard Deviation 0.382 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 43 | 0.17 millimole per Liter (mmol/L) | Standard Deviation 0.278 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration | Change From BL at Day 57 | 0.27 millimole per Liter (mmol/L) | Standard Deviation 0.726 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Baseline (BL) - Value at Visit | 19.94 unit per Liter (U/L) | Standard Deviation 10.951 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 2 | -0.50 unit per Liter (U/L) | Standard Deviation 1.862 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 4 | 0.13 unit per Liter (U/L) | Standard Deviation 1.996 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 8 | 0.19 unit per Liter (U/L) | Standard Deviation 3.016 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 15 | -0.13 unit per Liter (U/L) | Standard Deviation 10.379 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 29 | -0.13 unit per Liter (U/L) | Standard Deviation 3.202 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 43 | -0.13 unit per Liter (U/L) | Standard Deviation 3.845 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 57 | -0.25 unit per Liter (U/L) | Standard Deviation 4.712 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 71 | 0.13 unit per Liter (U/L) | Standard Deviation 5.353 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 85 | -0.69 unit per Liter (U/L) | Standard Deviation 3.79 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Change From BL at Day 113 | 1.75 unit per Liter (U/L) | Standard Deviation 5.756 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Post-BL Minimum Change From BL | -4.81 unit per Liter (U/L) | Standard Deviation 7.901 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration | Post-BL Maximum Change From BL | 5.94 unit per Liter (U/L) | Standard Deviation 6.319 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 2 | -10.13 unit per Liter (U/L) | Standard Deviation 13.495 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Baseline (BL) - Value at Visit | 153.75 unit per Liter (U/L) | Standard Deviation 22.228 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 4 | -0.19 unit per Liter (U/L) | Standard Deviation 15.246 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 8 | 3.19 unit per Liter (U/L) | Standard Deviation 11.053 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 15 | 3.19 unit per Liter (U/L) | Standard Deviation 17.505 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 29 | 6.56 unit per Liter (U/L) | Standard Deviation 17.397 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 43 | 2.38 unit per Liter (U/L) | Standard Deviation 11.73 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 57 | 1.69 unit per Liter (U/L) | Standard Deviation 11.247 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 71 | 0.19 unit per Liter (U/L) | Standard Deviation 15.003 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 85 | 2.63 unit per Liter (U/L) | Standard Deviation 16.536 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Change From BL at Day 113 | -0.38 unit per Liter (U/L) | Standard Deviation 16.141 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Post-BL Minimum Change From BL | -16.06 unit per Liter (U/L) | Standard Deviation 11.168 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration | Post-BL Maximum Change From BL | 22.00 unit per Liter (U/L) | Standard Deviation 13.765 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 113 | -0.05 millimole per Liter (mmol/L) | Standard Deviation 0.371 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Baseline (BL) - Value at Visit | 4.27 millimole per Liter (mmol/L) | Standard Deviation 0.351 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 2 | 0.11 millimole per Liter (mmol/L) | Standard Deviation 0.401 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 4 | -0.03 millimole per Liter (mmol/L) | Standard Deviation 0.307 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 8 | 0.12 millimole per Liter (mmol/L) | Standard Deviation 0.316 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 15 | -0.02 millimole per Liter (mmol/L) | Standard Deviation 0.351 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 29 | -0.05 millimole per Liter (mmol/L) | Standard Deviation 0.337 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 43 | 0.06 millimole per Liter (mmol/L) | Standard Deviation 0.366 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 57 | 0.05 millimole per Liter (mmol/L) | Standard Deviation 0.507 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 71 | -0.04 millimole per Liter (mmol/L) | Standard Deviation 0.32 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Change From BL at Day 85 | -0.19 millimole per Liter (mmol/L) | Standard Deviation 0.317 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Post-BL Minimum Change From BL | -0.47 millimole per Liter (mmol/L) | Standard Deviation 0.311 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration | Post-BL Maximum Change From BL | 0.45 millimole per Liter (mmol/L) | Standard Deviation 0.248 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Baseline (BL) - Value at Visit | 16.63 unit per Liter (U/L) | Standard Deviation 2.918 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 2 | -0.81 unit per Liter (U/L) | Standard Deviation 2.007 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 4 | 1.13 unit per Liter (U/L) | Standard Deviation 2.941 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 8 | 0.69 unit per Liter (U/L) | Standard Deviation 3.24 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 15 | 0.19 unit per Liter (U/L) | Standard Deviation 3.728 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 29 | 1.13 unit per Liter (U/L) | Standard Deviation 3.304 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 43 | -0.25 unit per Liter (U/L) | Standard Deviation 1.77 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 57 | 0.69 unit per Liter (U/L) | Standard Deviation 3.219 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 71 | -0.31 unit per Liter (U/L) | Standard Deviation 2.798 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 85 | -0.63 unit per Liter (U/L) | Standard Deviation 2.778 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Change From BL at Day 113 | -0.19 unit per Liter (U/L) | Standard Deviation 3.92 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Post-BL Minimum Change From BL | -2.94 unit per Liter (U/L) | Standard Deviation 2.594 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration | Post-BL Maximum Change From BL | 4.75 unit per Liter (U/L) | Standard Deviation 3.416 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 2 | -0.31 unit per Liter (U/L) | Standard Deviation 2.798 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Post-BL Maximum Change From BL | 8.25 unit per Liter (U/L) | Standard Deviation 6.943 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Baseline (BL) - Value at Visit | 15.63 unit per Liter (U/L) | Standard Deviation 6.449 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 4 | 2.19 unit per Liter (U/L) | Standard Deviation 5.419 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 8 | 2.19 unit per Liter (U/L) | Standard Deviation 3.953 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 15 | -0.13 unit per Liter (U/L) | Standard Deviation 7.702 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 29 | 0.25 unit per Liter (U/L) | Standard Deviation 6.34 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 43 | -1.69 unit per Liter (U/L) | Standard Deviation 5.199 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 57 | -1.13 unit per Liter (U/L) | Standard Deviation 6.49 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 71 | -0.50 unit per Liter (U/L) | Standard Deviation 6.408 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 85 | -1.13 unit per Liter (U/L) | Standard Deviation 5.56 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Change From BL at Day 113 | -0.63 unit per Liter (U/L) | Standard Deviation 7.571 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration | Post-BL Minimum Change From BL | -5.13 unit per Liter (U/L) | Standard Deviation 6.206 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Baseline (BL) - Value at Visit | 140.56 millimole per Liter (mmol/L) | Standard Deviation 1.59 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 2 | -0.44 millimole per Liter (mmol/L) | Standard Deviation 1.931 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 4 | -0.44 millimole per Liter (mmol/L) | Standard Deviation 1.931 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 8 | -0.06 millimole per Liter (mmol/L) | Standard Deviation 2.594 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 15 | 0.25 millimole per Liter (mmol/L) | Standard Deviation 1.612 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 29 | -1.75 millimole per Liter (mmol/L) | Standard Deviation 2.671 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 43 | -0.13 millimole per Liter (mmol/L) | Standard Deviation 2.062 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 57 | 0.06 millimole per Liter (mmol/L) | Standard Deviation 2.932 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 71 | -1.25 millimole per Liter (mmol/L) | Standard Deviation 3.13 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 85 | -0.38 millimole per Liter (mmol/L) | Standard Deviation 2.705 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Change From BL at Day 113 | -0.13 millimole per Liter (mmol/L) | Standard Deviation 1.893 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Post-BL Minimum Change From BL | -3.38 millimole per Liter (mmol/L) | Standard Deviation 2.391 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration | Post-BL Maximum Change From BL | 2.31 millimole per Liter (mmol/L) | Standard Deviation 1.922 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Post-BL Minimum Change From BL | -3.95 gram per Liter (g/L) | Standard Deviation 3.488 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Baseline (BL) - Value at Visit | 70.55 gram per Liter (g/L) | Standard Deviation 3.485 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 2 | -0.18 gram per Liter (g/L) | Standard Deviation 4.502 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 4 | 2.69 gram per Liter (g/L) | Standard Deviation 3.711 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 8 | 1.36 gram per Liter (g/L) | Standard Deviation 3.458 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 15 | 0.81 gram per Liter (g/L) | Standard Deviation 3.837 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 29 | 1.03 gram per Liter (g/L) | Standard Deviation 2.632 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 43 | -0.03 gram per Liter (g/L) | Standard Deviation 2.452 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 57 | -1.41 gram per Liter (g/L) | Standard Deviation 3.502 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 71 | 0.17 gram per Liter (g/L) | Standard Deviation 3.72 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 85 | -1.13 gram per Liter (g/L) | Standard Deviation 4.063 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Change From BL at Day 113 | 0.71 gram per Liter (g/L) | Standard Deviation 3.779 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration | Post-BL Maximum Change From BL | 4.54 gram per Liter (g/L) | Standard Deviation 2.451 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Baseline (BL) - Value at Visit | 323.44 micromole per Liter (μmol/L) | Standard Deviation 52.081 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 2 | 21.38 micromole per Liter (μmol/L) | Standard Deviation 28.329 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 4 | -15.06 micromole per Liter (μmol/L) | Standard Deviation 38.979 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 8 | -2.88 micromole per Liter (μmol/L) | Standard Deviation 35.955 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 15 | 2.44 micromole per Liter (μmol/L) | Standard Deviation 49.536 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 29 | 2.31 micromole per Liter (μmol/L) | Standard Deviation 32.946 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 43 | 9.63 micromole per Liter (μmol/L) | Standard Deviation 45.045 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 57 | 9.38 micromole per Liter (μmol/L) | Standard Deviation 53.799 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 85 | 19.25 micromole per Liter (μmol/L) | Standard Deviation 50.464 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 113 | 34.56 micromole per Liter (μmol/L) | Standard Deviation 62.406 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Post-BL Minimum Change From BL | -39.94 micromole per Liter (μmol/L) | Standard Deviation 25.87 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Post-BL Maximum Change From BL | 79.25 micromole per Liter (μmol/L) | Standard Deviation 62.143 |
| Emicizumab | Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration | Change From BL at Day 71 | 33.81 micromole per Liter (μmol/L) | Standard Deviation 84.44 |
Secondary
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Baseline (BL) - Value at Visit | 31.63 second | Standard Deviation 2.111 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Change From BL at Day 2 | -2.79 second | Standard Deviation 1.626 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Change From BL at Day 11 | -4.82 second | Standard Deviation 1.466 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Change From BL at Day 29 | -3.63 second | Standard Deviation 1.643 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Change From BL at Day 57 | -2.14 second | Standard Deviation 1.177 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Change From BL at Day 113 | -0.70 second | Standard Deviation 1.517 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Post-BL Minimum Change From BL | -4.83 second | Standard Deviation 1.482 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time | Post-BL Maximum Change From BL | -0.56 second | Standard Deviation 1.463 |
Secondary
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57 and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Baseline (BL) - Value at Visit | 2.43 gram per Liter (g/L) | Standard Deviation 0.41 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Change From BL at Day 2 | 0.18 gram per Liter (g/L) | Standard Deviation 0.281 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Change From BL at Day 11 | 0.28 gram per Liter (g/L) | Standard Deviation 0.314 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Change From BL at Day 29 | 0.11 gram per Liter (g/L) | Standard Deviation 0.312 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Change From BL at Day 57 | 0.05 gram per Liter (g/L) | Standard Deviation 0.285 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Change From BL at Day 113 | 0.07 gram per Liter (g/L) | Standard Deviation 0.438 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Post-BL Minimum Change From BL | -0.17 gram per Liter (g/L) | Standard Deviation 0.222 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration | Post-BL Maximum Change From BL | 0.53 gram per Liter (g/L) | Standard Deviation 0.29 |
Secondary
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57 and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Baseline (BL) - Value at Visit | 11.33 second | Standard Deviation 0.685 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Change From BL at Day 2 | 0.00 second | Standard Deviation 0.502 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Change From BL at Day 11 | -0.09 second | Standard Deviation 0.463 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Change From BL at Day 29 | -0.18 second | Standard Deviation 0.455 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Change From BL at Day 57 | -0.18 second | Standard Deviation 0.508 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Change From BL at Day 113 | -0.17 second | Standard Deviation 0.632 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Post-BL Minimum Change From BL | -0.58 second | Standard Deviation 0.457 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time | Post-BL Maximum Change From BL | 0.37 second | Standard Deviation 0.48 |
Secondary
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR)
The INR is a standardized measure of the prothrombin time. Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57 and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Baseline (BL) - Value at Visit | 1.06 INR of prothrombin time (sec/sec) | Standard Deviation 0.062 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Change From BL at Day 2 | 0.00 INR of prothrombin time (sec/sec) | Standard Deviation 0.044 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Change From BL at Day 11 | -0.01 INR of prothrombin time (sec/sec) | Standard Deviation 0.043 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Change From BL at Day 29 | -0.02 INR of prothrombin time (sec/sec) | Standard Deviation 0.042 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Change From BL at Day 57 | -0.02 INR of prothrombin time (sec/sec) | Standard Deviation 0.046 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Post-BL Minimum Change From BL | -0.05 INR of prothrombin time (sec/sec) | Standard Deviation 0.042 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Post-BL Maximum Change From BL | 0.03 INR of prothrombin time (sec/sec) | Standard Deviation 0.043 |
| Emicizumab | Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR) | Change From BL at Day 113 | -0.02 INR of prothrombin time (sec/sec) | Standard Deviation 0.057 |
Secondary
Change From Baseline in ECG Results by Timepoint: PR Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Baseline (BL) - Value at Visit | 160.21 millisecond | Standard Deviation 16.023 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 2 | 0.81 millisecond | Standard Deviation 12.767 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 4 | -0.10 millisecond | Standard Deviation 9.229 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 6 | -1.90 millisecond | Standard Deviation 9.998 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 8 | -4.13 millisecond | Standard Deviation 12.692 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 15 | -1.73 millisecond | Standard Deviation 14.577 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 29 | -4.10 millisecond | Standard Deviation 10.197 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 43 | -1.79 millisecond | Standard Deviation 13.85 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 57 | -1.52 millisecond | Standard Deviation 10.352 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 71 | -1.29 millisecond | Standard Deviation 9.008 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 85 | -5.02 millisecond | Standard Deviation 12.45 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Change From BL at Day 113 | -1.52 millisecond | Standard Deviation 6.576 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Post-BL Minimum Change From BL | -15.21 millisecond | Standard Deviation 11.517 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: PR Duration | Post-BL Maximum Change From BL | 8.83 millisecond | Standard Deviation 9 |
Secondary
Change From Baseline in ECG Results by Timepoint: QRS Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Baseline (BL) - Value at Visit | 98.15 millisecond | Standard Deviation 9.074 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 2 | 0.75 millisecond | Standard Deviation 5.514 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 4 | 1.40 millisecond | Standard Deviation 3.077 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 6 | -0.71 millisecond | Standard Deviation 3.052 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 8 | -0.48 millisecond | Standard Deviation 4.169 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 15 | -0.40 millisecond | Standard Deviation 3.221 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 29 | -1.00 millisecond | Standard Deviation 5.594 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 43 | -0.65 millisecond | Standard Deviation 2.327 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 57 | -1.54 millisecond | Standard Deviation 2.494 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 71 | 0.77 millisecond | Standard Deviation 3.429 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 85 | -0.46 millisecond | Standard Deviation 3.494 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Change From BL at Day 113 | -0.92 millisecond | Standard Deviation 3.782 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Post-BL Minimum Change From BL | -4.19 millisecond | Standard Deviation 3.592 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QRS Duration | Post-BL Maximum Change From BL | 4.50 millisecond | Standard Deviation 3.475 |
Secondary
Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula)
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Baseline (BL) - Value at Visit | 398.04 millisecond | Standard Deviation 19.275 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 2 | -3.81 millisecond | Standard Deviation 10.367 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 4 | -2.29 millisecond | Standard Deviation 8.759 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 6 | 7.33 millisecond | Standard Deviation 8.549 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 8 | 10.08 millisecond | Standard Deviation 13.968 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 15 | 10.50 millisecond | Standard Deviation 10.87 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 29 | 12.69 millisecond | Standard Deviation 12.031 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 43 | 8.81 millisecond | Standard Deviation 13.699 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 57 | 12.00 millisecond | Standard Deviation 9.509 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 71 | 12.25 millisecond | Standard Deviation 12.194 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 85 | 15.27 millisecond | Standard Deviation 13.029 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Change From BL at Day 113 | 12.08 millisecond | Standard Deviation 13.229 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Post-BL Minimum Change From BL | -7.27 millisecond | Standard Deviation 8.817 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula) | Post-BL Maximum Change From BL | 24.23 millisecond | Standard Deviation 11.165 |
Secondary
Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula)
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Baseline (BL) - Value at Visit | 397.90 millisecond | Standard Deviation 20.554 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 2 | -2.98 millisecond | Standard Deviation 8.968 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 4 | -4.94 millisecond | Standard Deviation 9.038 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 6 | 1.21 millisecond | Standard Deviation 9.739 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 8 | 1.31 millisecond | Standard Deviation 9.708 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 15 | 3.56 millisecond | Standard Deviation 10.109 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 29 | 4.33 millisecond | Standard Deviation 8.384 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 43 | 1.69 millisecond | Standard Deviation 8.274 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 57 | 5.31 millisecond | Standard Deviation 8.893 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 71 | 6.40 millisecond | Standard Deviation 11.109 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 85 | 6.71 millisecond | Standard Deviation 12.243 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Change From BL at Day 113 | 4.81 millisecond | Standard Deviation 12.931 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Post-BL Minimum Change From BL | -8.21 millisecond | Standard Deviation 8.174 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula) | Post-BL Maximum Change From BL | 14.77 millisecond | Standard Deviation 10.366 |
Secondary
Change From Baseline in ECG Results by Timepoint: QT Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Baseline (BL) - Value at Visit | 398.31 millisecond | Standard Deviation 27.123 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 2 | -1.35 millisecond | Standard Deviation 11.351 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 4 | -10.00 millisecond | Standard Deviation 13.856 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 6 | -12.31 millisecond | Standard Deviation 19.791 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 8 | -15.69 millisecond | Standard Deviation 15.037 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 15 | -9.90 millisecond | Standard Deviation 13.857 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 29 | -11.83 millisecond | Standard Deviation 19.322 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 43 | -11.96 millisecond | Standard Deviation 13.863 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 57 | -7.56 millisecond | Standard Deviation 16.498 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 71 | -4.94 millisecond | Standard Deviation 19.933 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 85 | -9.75 millisecond | Standard Deviation 23.282 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Change From BL at Day 113 | -9.21 millisecond | Standard Deviation 20.349 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Post-BL Minimum Change From BL | -30.35 millisecond | Standard Deviation 14.706 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: QT Duration | Post-BL Maximum Change From BL | 9.60 millisecond | Standard Deviation 12.064 |
Secondary
Change From Baseline in ECG Results by Timepoint: RR Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Baseline (BL) - Value at Visit | 1001.06 millisecond | Standard Deviation 93.082 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 2 | 12.73 millisecond | Standard Deviation 66.16 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 4 | -36.04 millisecond | Standard Deviation 60.573 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 6 | -94.79 millisecond | Standard Deviation 99.788 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 8 | -119.67 millisecond | Standard Deviation 106.349 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 15 | -96.56 millisecond | Standard Deviation 67.805 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 29 | -114.08 millisecond | Standard Deviation 126.347 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 43 | -97.98 millisecond | Standard Deviation 109.011 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 57 | -92.98 millisecond | Standard Deviation 87.99 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 71 | -82.29 millisecond | Standard Deviation 112.001 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 85 | -115.87 millisecond | Standard Deviation 122.073 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Change From BL at Day 113 | -101.02 millisecond | Standard Deviation 98.269 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Post-BL Minimum Change From BL | -205.40 millisecond | Standard Deviation 91.026 |
| Emicizumab | Change From Baseline in ECG Results by Timepoint: RR Duration | Post-BL Maximum Change From BL | 40.25 millisecond | Standard Deviation 49.296 |
Secondary
Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 8 | 8.63 beats per minute | Standard Deviation 7.574 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Baseline (BL) - Value at Visit | 59.90 beats per minute | Standard Deviation 5.588 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 2 | -0.67 beats per minute | Standard Deviation 3.656 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 4 | 2.50 beats per minute | Standard Deviation 3.978 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 6 | 6.52 beats per minute | Standard Deviation 6.491 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 15 | 6.58 beats per minute | Standard Deviation 4.25 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 29 | 8.10 beats per minute | Standard Deviation 8.675 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 43 | 6.96 beats per minute | Standard Deviation 7.946 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 57 | 6.42 beats per minute | Standard Deviation 5.793 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 71 | 5.42 beats per minute | Standard Deviation 6.976 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 85 | 8.25 beats per minute | Standard Deviation 8.234 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Change From BL at Day 113 | 7.15 beats per minute | Standard Deviation 6.442 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Post-BL Minimum Change From BL | -2.21 beats per minute | Standard Deviation 2.638 |
| Emicizumab | Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate | Post-BL Maximum Change From BL | 15.52 beats per minute | Standard Deviation 6.259 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Baseline (BL) - Value at Visit | 0.02 *10^9 cells per Liter | Standard Deviation 0.018 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 2 | 0.00 *10^9 cells per Liter | Standard Deviation 0.013 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 4 | 0.01 *10^9 cells per Liter | Standard Deviation 0.018 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 8 | 0.01 *10^9 cells per Liter | Standard Deviation 0.029 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 15 | 0.01 *10^9 cells per Liter | Standard Deviation 0.022 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 29 | 0.01 *10^9 cells per Liter | Standard Deviation 0.014 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 43 | 0.01 *10^9 cells per Liter | Standard Deviation 0.023 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 57 | 0.01 *10^9 cells per Liter | Standard Deviation 0.016 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 71 | 0.01 *10^9 cells per Liter | Standard Deviation 0.022 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 85 | 0.00 *10^9 cells per Liter | Standard Deviation 0.021 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Change From BL at Day 113 | 0.01 *10^9 cells per Liter | Standard Deviation 0.019 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Post-BL Minimum Change From BL | -0.01 *10^9 cells per Liter | Standard Deviation 0.016 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count | Post-BL Maximum Change From BL | 0.03 *10^9 cells per Liter | Standard Deviation 0.021 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 2 | 0.02 *10^9 cells per Liter | Standard Deviation 0.063 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 15 | -0.01 *10^9 cells per Liter | Standard Deviation 0.074 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 29 | -0.01 *10^9 cells per Liter | Standard Deviation 0.055 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 57 | -0.02 *10^9 cells per Liter | Standard Deviation 0.055 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Baseline (BL) - Value at Visit | 0.14 *10^9 cells per Liter | Standard Deviation 0.122 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 4 | 0.01 *10^9 cells per Liter | Standard Deviation 0.066 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 8 | 0.00 *10^9 cells per Liter | Standard Deviation 0.066 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 43 | 0.00 *10^9 cells per Liter | Standard Deviation 0.101 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 71 | 0.01 *10^9 cells per Liter | Standard Deviation 0.102 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 85 | -0.02 *10^9 cells per Liter | Standard Deviation 0.063 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Change From BL at Day 113 | 0.02 *10^9 cells per Liter | Standard Deviation 0.121 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Post-BL Minimum Change From BL | -0.06 *10^9 cells per Liter | Standard Deviation 0.08 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count | Post-BL Maximum Change From BL | 0.07 *10^9 cells per Liter | Standard Deviation 0.085 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin
Erythrocyte mean corpuscular hemoglobin (MCH) is a measure of the average amount of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Baseline (BL) - Value at Visit | 30.84 picogram per cell | Standard Deviation 1.815 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 2 | 0.23 picogram per cell | Standard Deviation 0.467 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 4 | 0.09 picogram per cell | Standard Deviation 0.473 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 8 | 0.26 picogram per cell | Standard Deviation 0.594 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 15 | -0.12 picogram per cell | Standard Deviation 0.472 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 29 | 0.04 picogram per cell | Standard Deviation 0.507 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 43 | 0.48 picogram per cell | Standard Deviation 0.493 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 57 | 0.28 picogram per cell | Standard Deviation 0.567 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 71 | 0.40 picogram per cell | Standard Deviation 0.625 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 85 | 0.39 picogram per cell | Standard Deviation 0.738 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Change From BL at Day 113 | 0.51 picogram per cell | Standard Deviation 0.933 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Post-BL Minimum Change From BL | -0.40 picogram per cell | Standard Deviation 0.412 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin | Post-BL Maximum Change From BL | 1.24 picogram per cell | Standard Deviation 0.73 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration
Erythrocyte mean corpuscular hemoglobin concentration (MCHC) is a measure of the average concentration of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Baseline (BL) - Value at Visit | 343.25 gram per Liter (g/L) | Standard Deviation 8.79 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 2 | 3.13 gram per Liter (g/L) | Standard Deviation 6.438 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 4 | 1.00 gram per Liter (g/L) | Standard Deviation 7.239 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 8 | 2.31 gram per Liter (g/L) | Standard Deviation 5.606 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 15 | -4.56 gram per Liter (g/L) | Standard Deviation 6.562 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 29 | 0.69 gram per Liter (g/L) | Standard Deviation 6.019 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 43 | 0.13 gram per Liter (g/L) | Standard Deviation 8.853 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 57 | -0.75 gram per Liter (g/L) | Standard Deviation 8.798 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 71 | 1.25 gram per Liter (g/L) | Standard Deviation 6.372 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 85 | 1.13 gram per Liter (g/L) | Standard Deviation 8.277 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Change From BL at Day 113 | 3.25 gram per Liter (g/L) | Standard Deviation 8.004 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Post-BL Minimum Change From BL | -9.00 gram per Liter (g/L) | Standard Deviation 6.143 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration | Post-BL Maximum Change From BL | 10.56 gram per Liter (g/L) | Standard Deviation 6.099 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume
Erythrocyte mean corpuscular volume is a measure of the average volume of a red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 4 | 0.01 femtoliter (fL) | Standard Deviation 0.954 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 8 | 0.21 femtoliter (fL) | Standard Deviation 1.138 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 15 | 0.88 femtoliter (fL) | Standard Deviation 1.036 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 85 | 0.86 femtoliter (fL) | Standard Deviation 1.656 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Baseline (BL) - Value at Visit | 89.79 femtoliter (fL) | Standard Deviation 3.689 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 2 | -0.14 femtoliter (fL) | Standard Deviation 1.172 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 29 | -0.08 femtoliter (fL) | Standard Deviation 0.849 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 43 | 1.41 femtoliter (fL) | Standard Deviation 1.67 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 57 | 1.04 femtoliter (fL) | Standard Deviation 1.488 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 71 | 0.85 femtoliter (fL) | Standard Deviation 1.498 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Change From BL at Day 113 | 0.62 femtoliter (fL) | Standard Deviation 1.775 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Post-BL Minimum Change From BL | -1.17 femtoliter (fL) | Standard Deviation 0.899 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume | Post-BL Maximum Change From BL | 2.34 femtoliter (fL) | Standard Deviation 1.138 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1)
Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 4 | 0.01 ratio of RBCs in blood (L/L) | Standard Deviation 0.024 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 113 | 0.01 ratio of RBCs in blood (L/L) | Standard Deviation 0.029 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Post-BL Minimum Change From BL | -0.02 ratio of RBCs in blood (L/L) | Standard Deviation 0.02 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Baseline (BL) - Value at Visit | 0.44 ratio of RBCs in blood (L/L) | Standard Deviation 0.02 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 2 | 0.01 ratio of RBCs in blood (L/L) | Standard Deviation 0.032 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 8 | 0.00 ratio of RBCs in blood (L/L) | Standard Deviation 0.023 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 15 | 0.00 ratio of RBCs in blood (L/L) | Standard Deviation 0.018 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 29 | -0.01 ratio of RBCs in blood (L/L) | Standard Deviation 0.019 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 43 | 0.00 ratio of RBCs in blood (L/L) | Standard Deviation 0.025 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 57 | -0.01 ratio of RBCs in blood (L/L) | Standard Deviation 0.027 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 71 | 0.00 ratio of RBCs in blood (L/L) | Standard Deviation 0.027 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Change From BL at Day 85 | 0.00 ratio of RBCs in blood (L/L) | Standard Deviation 0.029 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1) | Post-BL Maximum Change From BL | 0.02 ratio of RBCs in blood (L/L) | Standard Deviation 0.022 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Baseline (BL) - Value at Visit | 149.38 gram per Liter (g/L) | Standard Deviation 8.065 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 2 | 3.94 gram per Liter (g/L) | Standard Deviation 10.056 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 4 | 5.38 gram per Liter (g/L) | Standard Deviation 7.571 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 8 | 0.88 gram per Liter (g/L) | Standard Deviation 6.551 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 15 | -2.50 gram per Liter (g/L) | Standard Deviation 6.439 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 29 | -2.50 gram per Liter (g/L) | Standard Deviation 7.062 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 43 | -0.56 gram per Liter (g/L) | Standard Deviation 6.345 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 57 | -1.94 gram per Liter (g/L) | Standard Deviation 8.33 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 71 | 0.94 gram per Liter (g/L) | Standard Deviation 7.505 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 85 | 0.88 gram per Liter (g/L) | Standard Deviation 7.881 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Change From BL at Day 113 | 3.19 gram per Liter (g/L) | Standard Deviation 9.086 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Post-BL Minimum Change From BL | -8.13 gram per Liter (g/L) | Standard Deviation 6.076 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration | Post-BL Maximum Change From BL | 9.63 gram per Liter (g/L) | Standard Deviation 5.608 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 4 | 0.37 *10^9 cells per Liter | Standard Deviation 0.78 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 8 | 0.04 *10^9 cells per Liter | Standard Deviation 0.595 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 15 | -0.09 *10^9 cells per Liter | Standard Deviation 0.599 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 29 | -0.01 *10^9 cells per Liter | Standard Deviation 0.422 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 43 | 0.07 *10^9 cells per Liter | Standard Deviation 0.47 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 57 | 0.01 *10^9 cells per Liter | Standard Deviation 0.464 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 71 | 0.06 *10^9 cells per Liter | Standard Deviation 0.372 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 85 | 0.02 *10^9 cells per Liter | Standard Deviation 0.397 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 113 | 0.04 *10^9 cells per Liter | Standard Deviation 0.585 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Post-BL Minimum Change From BL | -0.35 *10^9 cells per Liter | Standard Deviation 0.447 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Post-BL Maximum Change From BL | 0.61 *10^9 cells per Liter | Standard Deviation 0.633 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Baseline (BL) - Value at Visit | 1.65 *10^9 cells per Liter | Standard Deviation 0.691 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count | Change From BL at Day 2 | 0.10 *10^9 cells per Liter | Standard Deviation 0.381 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 113 | -0.02 *10^9 cells per Liter | Standard Deviation 0.112 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Baseline (BL) - Value at Visit | 0.35 *10^9 cells per Liter | Standard Deviation 0.087 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 2 | -0.04 *10^9 cells per Liter | Standard Deviation 0.098 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 4 | -0.03 *10^9 cells per Liter | Standard Deviation 0.111 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 8 | -0.04 *10^9 cells per Liter | Standard Deviation 0.1 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 15 | -0.01 *10^9 cells per Liter | Standard Deviation 0.136 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 29 | -0.01 *10^9 cells per Liter | Standard Deviation 0.112 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 43 | -0.03 *10^9 cells per Liter | Standard Deviation 0.082 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 57 | -0.04 *10^9 cells per Liter | Standard Deviation 0.091 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 71 | -0.03 *10^9 cells per Liter | Standard Deviation 0.075 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Change From BL at Day 85 | -0.04 *10^9 cells per Liter | Standard Deviation 0.096 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Post-BL Minimum Change From BL | -0.10 *10^9 cells per Liter | Standard Deviation 0.08 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count | Post-BL Maximum Change From BL | 0.11 *10^9 cells per Liter | Standard Deviation 0.112 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 57 | 10.94 *10^9 cells per Liter | Standard Deviation 18.908 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 113 | 2.94 *10^9 cells per Liter | Standard Deviation 23.778 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Baseline (BL) - Value at Visit | 231.50 *10^9 cells per Liter | Standard Deviation 47.88 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 2 | -2.13 *10^9 cells per Liter | Standard Deviation 25.469 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 4 | -3.69 *10^9 cells per Liter | Standard Deviation 22.33 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 8 | 0.06 *10^9 cells per Liter | Standard Deviation 20.612 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 15 | 7.25 *10^9 cells per Liter | Standard Deviation 23.023 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 29 | 12.50 *10^9 cells per Liter | Standard Deviation 26.967 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 43 | 9.88 *10^9 cells per Liter | Standard Deviation 30.265 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 71 | 5.50 *10^9 cells per Liter | Standard Deviation 34.131 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Change From BL at Day 85 | 0.50 *10^9 cells per Liter | Standard Deviation 30.542 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Post-BL Minimum Change From BL | -27.31 *10^9 cells per Liter | Standard Deviation 17.984 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count | Post-BL Maximum Change From BL | 36.50 *10^9 cells per Liter | Standard Deviation 26.473 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Baseline (BL) - Value at Visit | 4.86 *10^12 cells per Liter | Standard Deviation 0.304 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 2 | 0.09 *10^12 cells per Liter | Standard Deviation 0.327 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 4 | 0.16 *10^12 cells per Liter | Standard Deviation 0.247 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 8 | -0.02 *10^12 cells per Liter | Standard Deviation 0.235 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 15 | -0.07 *10^12 cells per Liter | Standard Deviation 0.196 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 29 | -0.09 *10^12 cells per Liter | Standard Deviation 0.209 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 43 | -0.09 *10^12 cells per Liter | Standard Deviation 0.226 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 57 | -0.11 *10^12 cells per Liter | Standard Deviation 0.289 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 71 | -0.04 *10^12 cells per Liter | Standard Deviation 0.266 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 85 | -0.04 *10^12 cells per Liter | Standard Deviation 0.334 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Change From BL at Day 113 | 0.02 *10^12 cells per Liter | Standard Deviation 0.337 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Post-BL Minimum Change From BL | -0.31 *10^12 cells per Liter | Standard Deviation 0.242 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count | Post-BL Maximum Change From BL | 0.26 *10^12 cells per Liter | Standard Deviation 0.213 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Post-BL Minimum Change From BL | -0.86 *10^9 cells per Liter | Standard Deviation 0.708 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Baseline (BL) - Value at Visit | 3.57 *10^9 cells per Liter | Standard Deviation 0.869 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 2 | -0.24 *10^9 cells per Liter | Standard Deviation 0.959 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 4 | 0.12 *10^9 cells per Liter | Standard Deviation 0.95 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 8 | -0.19 *10^9 cells per Liter | Standard Deviation 0.809 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 15 | 0.19 *10^9 cells per Liter | Standard Deviation 1.043 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 29 | 0.23 *10^9 cells per Liter | Standard Deviation 1.063 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 43 | 0.06 *10^9 cells per Liter | Standard Deviation 0.961 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 57 | -0.24 *10^9 cells per Liter | Standard Deviation 0.758 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 71 | -0.06 *10^9 cells per Liter | Standard Deviation 0.824 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 85 | -0.31 *10^9 cells per Liter | Standard Deviation 0.686 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Change From BL at Day 113 | -0.27 *10^9 cells per Liter | Standard Deviation 0.695 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count | Post-BL Maximum Change From BL | 1.16 *10^9 cells per Liter | Standard Deviation 0.968 |
Secondary
Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Baseline (BL) - Value at Visit | 5.74 *10^9 cells per Liter | Standard Deviation 1.565 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 2 | -0.17 *10^9 cells per Liter | Standard Deviation 1.213 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 4 | 0.33 *10^9 cells per Liter | Standard Deviation 1.285 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 8 | -0.20 *10^9 cells per Liter | Standard Deviation 1.222 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 15 | 0.09 *10^9 cells per Liter | Standard Deviation 1.4 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 29 | 0.21 *10^9 cells per Liter | Standard Deviation 1.026 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 43 | 0.11 *10^9 cells per Liter | Standard Deviation 1.162 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 57 | -0.27 *10^9 cells per Liter | Standard Deviation 1.125 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 71 | -0.01 *10^9 cells per Liter | Standard Deviation 1.006 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 85 | -0.34 *10^9 cells per Liter | Standard Deviation 1.064 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Change From BL at Day 113 | -0.24 *10^9 cells per Liter | Standard Deviation 1.332 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Post-BL Minimum Change From BL | -1.07 *10^9 cells per Liter | Standard Deviation 1.071 |
| Emicizumab | Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count | Post-BL Maximum Change From BL | 1.29 *10^9 cells per Liter | Standard Deviation 0.997 |
Secondary
Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Baseline (BL) - Value at Visit | 66.1 millimeters of mercury (mmHg) | Standard Deviation 5.77 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 2 | -0.8 millimeters of mercury (mmHg) | Standard Deviation 4.99 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 4 | 2.4 millimeters of mercury (mmHg) | Standard Deviation 5.21 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 6 | 3.2 millimeters of mercury (mmHg) | Standard Deviation 5.43 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 8 | 2.7 millimeters of mercury (mmHg) | Standard Deviation 5.41 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 11 | 1.4 millimeters of mercury (mmHg) | Standard Deviation 5.11 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 15 | 1.7 millimeters of mercury (mmHg) | Standard Deviation 4.24 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 22 | 3.2 millimeters of mercury (mmHg) | Standard Deviation 5.46 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 29 | 2.6 millimeters of mercury (mmHg) | Standard Deviation 5.67 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 36 | 1.3 millimeters of mercury (mmHg) | Standard Deviation 4.48 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 43 | 1.0 millimeters of mercury (mmHg) | Standard Deviation 5.18 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 50 | 3.4 millimeters of mercury (mmHg) | Standard Deviation 6.49 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 57 | -0.3 millimeters of mercury (mmHg) | Standard Deviation 4.64 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 71 | 1.1 millimeters of mercury (mmHg) | Standard Deviation 5.5 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 85 | 1.3 millimeters of mercury (mmHg) | Standard Deviation 4.52 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Change From BL at Day 113 | 1.3 millimeters of mercury (mmHg) | Standard Deviation 4.73 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Post-BL Minimum Change From BL | -5.2 millimeters of mercury (mmHg) | Standard Deviation 3.08 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure | Post-BL Maximum Change From BL | 8.1 millimeters of mercury (mmHg) | Standard Deviation 4.39 |
Secondary
Change From Baseline in Vital Signs by Timepoint: Pulse Rate
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 6 | 5.8 beats per minute | Standard Deviation 7.06 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 43 | 6.2 beats per minute | Standard Deviation 7.93 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Baseline (BL) - Value at Visit | 61.3 beats per minute | Standard Deviation 7.25 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 2 | -0.6 beats per minute | Standard Deviation 4.72 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 4 | 1.3 beats per minute | Standard Deviation 4.9 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 8 | 7.7 beats per minute | Standard Deviation 8.58 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 11 | 9.9 beats per minute | Standard Deviation 10.41 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 15 | 6.9 beats per minute | Standard Deviation 5.71 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 22 | 10.2 beats per minute | Standard Deviation 9.22 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 29 | 8.4 beats per minute | Standard Deviation 9.24 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 36 | 6.3 beats per minute | Standard Deviation 7.59 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 50 | 10.4 beats per minute | Standard Deviation 6.98 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 57 | 7.5 beats per minute | Standard Deviation 6.75 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 71 | 4.7 beats per minute | Standard Deviation 7.07 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 85 | 6.9 beats per minute | Standard Deviation 9.48 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Change From BL at Day 113 | 7.4 beats per minute | Standard Deviation 8.16 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Post-BL Minimum Change From BL | -3.1 beats per minute | Standard Deviation 4.46 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Pulse Rate | Post-BL Maximum Change From BL | 18.1 beats per minute | Standard Deviation 6.29 |
Secondary
Change From Baseline in Vital Signs by Timepoint: Respiratory Rate
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 29 | 0.2 breaths per minute | Standard Deviation 2.1 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Post-BL Minimum Change From BL | -3.0 breaths per minute | Standard Deviation 1.83 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Post-BL Maximum Change From BL | 2.7 breaths per minute | Standard Deviation 1.82 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Baseline (BL) - Value at Visit | 15.5 breaths per minute | Standard Deviation 1.51 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 2 | 0.6 breaths per minute | Standard Deviation 1.5 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 22 | -0.1 breaths per minute | Standard Deviation 2.6 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 4 | 0.3 breaths per minute | Standard Deviation 1.89 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 6 | -0.5 breaths per minute | Standard Deviation 2 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 8 | 0.8 breaths per minute | Standard Deviation 2.64 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 11 | 0.1 breaths per minute | Standard Deviation 2.55 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 15 | -0.1 breaths per minute | Standard Deviation 2.38 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 36 | 0.9 breaths per minute | Standard Deviation 1.54 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 43 | -0.3 breaths per minute | Standard Deviation 1.88 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 50 | 0.3 breaths per minute | Standard Deviation 2.32 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 57 | 0.2 breaths per minute | Standard Deviation 2.14 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 71 | -0.3 breaths per minute | Standard Deviation 2.02 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 85 | -0.4 breaths per minute | Standard Deviation 2.33 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Respiratory Rate | Change From BL at Day 113 | 0.4 breaths per minute | Standard Deviation 2.47 |
Secondary
Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Baseline (BL) - Value at Visit | 106.8 millimeters of mercury (mmHg) | Standard Deviation 7.26 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 2 | -1.6 millimeters of mercury (mmHg) | Standard Deviation 6.49 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 4 | 2.4 millimeters of mercury (mmHg) | Standard Deviation 5.82 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 6 | 3.6 millimeters of mercury (mmHg) | Standard Deviation 5.86 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 8 | 3.5 millimeters of mercury (mmHg) | Standard Deviation 8.16 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 11 | 5.1 millimeters of mercury (mmHg) | Standard Deviation 8.05 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 15 | 2.5 millimeters of mercury (mmHg) | Standard Deviation 4.97 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 22 | 4.8 millimeters of mercury (mmHg) | Standard Deviation 7.46 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 29 | 3.2 millimeters of mercury (mmHg) | Standard Deviation 8.84 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 36 | 2.2 millimeters of mercury (mmHg) | Standard Deviation 7.49 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 43 | 2.0 millimeters of mercury (mmHg) | Standard Deviation 5.97 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 50 | 5.2 millimeters of mercury (mmHg) | Standard Deviation 8.95 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 57 | 2.2 millimeters of mercury (mmHg) | Standard Deviation 7.19 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 71 | 1.2 millimeters of mercury (mmHg) | Standard Deviation 6.35 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 85 | 2.0 millimeters of mercury (mmHg) | Standard Deviation 8.68 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Change From BL at Day 113 | 1.1 millimeters of mercury (mmHg) | Standard Deviation 8.75 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Post-BL Minimum Change From BL | -4.6 millimeters of mercury (mmHg) | Standard Deviation 5.76 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure | Post-BL Maximum Change From BL | 10.9 millimeters of mercury (mmHg) | Standard Deviation 7.47 |
Secondary
Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary)
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 29 | -0.11 degrees Celsius (C) | Standard Deviation 0.433 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Baseline (BL) - Value at Visit | 36.20 degrees Celsius (C) | Standard Deviation 0.239 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 2 | -0.12 degrees Celsius (C) | Standard Deviation 0.281 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 4 | 0.01 degrees Celsius (C) | Standard Deviation 0.263 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 6 | -0.09 degrees Celsius (C) | Standard Deviation 0.532 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 8 | -0.08 degrees Celsius (C) | Standard Deviation 0.217 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 11 | -0.18 degrees Celsius (C) | Standard Deviation 0.414 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 15 | -0.06 degrees Celsius (C) | Standard Deviation 0.515 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 22 | -0.14 degrees Celsius (C) | Standard Deviation 0.352 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 36 | -0.19 degrees Celsius (C) | Standard Deviation 0.313 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 43 | -0.16 degrees Celsius (C) | Standard Deviation 0.318 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 50 | -0.16 degrees Celsius (C) | Standard Deviation 0.418 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 57 | 0.02 degrees Celsius (C) | Standard Deviation 0.382 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 71 | -0.09 degrees Celsius (C) | Standard Deviation 0.36 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 85 | -0.21 degrees Celsius (C) | Standard Deviation 0.26 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Change From BL at Day 113 | -0.02 degrees Celsius (C) | Standard Deviation 0.297 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Post-BL Minimum Change From BL | -0.61 degrees Celsius (C) | Standard Deviation 0.26 |
| Emicizumab | Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary) | Post-BL Maximum Change From BL | 0.43 degrees Celsius (C) | Standard Deviation 0.357 |
Secondary
Mean Residence Time (MRT) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Emicizumab | Mean Residence Time (MRT) of Emicizumab | 40.26 day | Standard Deviation 6.34 |
Secondary
Number of Participants by Test Results for Blood in Urine by Timepoint
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for blood in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 8, 29, 57, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Baseline | 0 (Absent) | 15 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Baseline | +1 (Trace) | 1 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Baseline | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Baseline | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 2 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 2 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 2 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 2 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 8 | 0 (Absent) | 15 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 8 | +1 (Trace) | 1 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 8 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 8 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 29 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 29 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 29 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 29 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 57 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 57 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 57 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 57 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 85 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 85 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 85 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 85 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 113 | 0 (Absent) | 15 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 113 | +1 (Trace) | 1 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 113 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Blood in Urine by Timepoint | Day 113 | +3/+4 (Strong Positive) | 0 Participants |
Secondary
Number of Participants by Test Results for Glucose in Urine by Timepoint
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for glucose in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 8, 29, 57, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 57 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 113 | +1 (Trace) | 1 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Baseline | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Baseline | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Baseline | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Baseline | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 2 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 2 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 2 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 2 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 8 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 8 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 8 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 8 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 29 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 29 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 29 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 29 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 57 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 57 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 57 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 85 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 85 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 85 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 85 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 113 | 0 (Absent) | 15 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 113 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Glucose in Urine by Timepoint | Day 113 | +3/+4 (Strong Positive) | 0 Participants |
Secondary
Number of Participants by Test Results for Protein in Urine by Timepoint
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for protein in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 8, 29, 57, 85, and 113
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Baseline | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Baseline | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Baseline | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Baseline | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 2 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 2 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 2 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 2 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 8 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 8 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 8 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 8 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 29 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 29 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 29 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 29 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 57 | 0 (Absent) | 15 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 57 | +1 (Trace) | 1 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 57 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 57 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 85 | 0 (Absent) | 15 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 85 | +1 (Trace) | 1 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 85 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 85 | +3/+4 (Strong Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 113 | 0 (Absent) | 16 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 113 | +1 (Trace) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 113 | +2 (Positive) | 0 Participants |
| Emicizumab | Number of Participants by Test Results for Protein in Urine by Timepoint | Day 113 | +3/+4 (Strong Positive) | 0 Participants |
Secondary
Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study
Participants were considered to be 'ADA Negative (Treatment Unaffected)' if baseline and all post-baseline samples were negative, or if they were ADA positive at baseline but did not have any post-baseline samples with a titer that was at least 4-fold greater than the titer of the baseline sample. 'Total ADA Positive' is the sum of all participants who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Induced)', those who were ADA negative at baseline and tested positive for ADA following study drug administration; and 'ADA Positive (Treatment Boosted)', those who were pre-dose ADA positive and had post-baseline samples with a titer that was at least 4-fold greater compared to the baseline measurement.
Time frame: Predose at Baseline (Day 1) and postdose on Days 57 and 113
Population: Includes participants with at least one predose and one postdose anti-drug antibody (ADA) assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Day 113 - ADA Positive | 1 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Baseline - ADA Positive | 0 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Baseline - ADA Negative | 16 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Day 57 - ADA Positive | 0 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Day 57 - ADA Negative | 16 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Day 113 - ADA Negative | 15 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Overall - ADA Negative (Treatment Unaffected) | 15 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Overall - Total ADA Positive (Induced + Boosted) | 1 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Overall - ADA Positive (Treatment Induced) | 1 Participants |
| Emicizumab | Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study | Overall - ADA Positive (Treatment Boosted) | 0 Participants |
Secondary
Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade
The WHO Toxicity Grading Scale was used for assessing adverse event severity. Any adverse event not specifically listed in the WHO Toxicity Grading Scale was assessed according to the following levels of severity: Grade 1 is mild; Grade 2 is moderate; Grade 3 is severe; and Grade 4 is life-threatening. Investigator text for adverse events were encoded using the Medical Dictionary for Regulatory Activities (MedDRA) version 21.1. After informed consent had been obtained but prior to initiation of study drug, only serious adverse events (SAEs) caused by a protocol-mandated intervention were to have been reported. After initiation of study drug, all adverse events, regardless of relationship to study drug, were to have been reported until the participant completed his last study visit. After this period, any SAEs believed to be related to prior study drug treatment were to be reported.
Time frame: From screening to study completion (20 weeks)
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | White blood cell count decreased - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Hypokalaemia - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Any Adverse Event - Any Grade | 14 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Any Adverse Event - Grade 1 | 7 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Any Adverse Event - Grade 2 | 7 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Any Adverse Event - Grade 3 | 0 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Any Adverse Event - Grade 4 | 0 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Leukopenia - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Diarrhoea - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Flatulence - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Mouth ulceration - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Toothache - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Conjunctivitis - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Gastroenteritis - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Pericoronitis - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Upper respiratory tract infection - Grade 1 | 3 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Upper respiratory tract infection - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Injury - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Road traffic accident - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Blood bilirubin increased - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Blood creatinine phosphokinase increased - Grade 1 | 4 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Blood triglycerides increased - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Blood uric acid increased - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | C-reactive protein increased - Grade 1 | 2 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | White blood cell count increased - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Hypertriglyceridaemia - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Cough - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Dry throat - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Nasal congestion - Grade 1 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Nasal obstruction - Grade 2 | 1 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Oropharyngeal pain - Grade 1 | 3 Participants |
| Emicizumab | Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade | Rash - Grade 1 | 1 Participants |
Secondary
Number of Participants With Concomitant Medications
The original terms recorded by the investigator for concomitant medications were standardized by the sponsor by assigning preferred terms. The duration of treatment with the concomitant medications ranged from 1 day to 5 days. Except for 1 participant who was treated during the in-clinic period (Days -1 to 4), all other concomitant medications were recorded during the ambulatory period (Days 6 to 113).
Time frame: From screening to study completion (20 weeks)
Population: The safety analysis population included all participants who had received any amount of study medication, whether prematurely withdrawn from the study or not, and with at least one post-baseline safety assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Emicizumab | Number of Participants With Concomitant Medications | Total Participants with at Least 1 Treatment | 6 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Analgesics - Paracetamol | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Antitrichomonal Agents - Tinidazole | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Cephalosporin Antibiotics - Cefadroxil | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Cephalosporin Antibiotics - Cefminox | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Herbal, Homeopathic, and Dietary Supplements | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Non-Steroidal Anti-Inflammatories - Pranoprofen | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Proton Pump Inhibitors - Pantoprazole | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Salicylates - Aspirin DL-Lysine | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Supplements - Potassium Chloride | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Supplements - Sodium Chloride | 1 Participants |
| Emicizumab | Number of Participants With Concomitant Medications | Vitamins and Minerals - Ascorbic Acid | 1 Participants |
Secondary
Number of Participants With Laboratory Test Abnormalities
The number of participants with a laboratory abnormality during treatment (numerator) is reported among the 'number analyzed' in the table below (denominator), which represents the number of participants without that abnormality at baseline (last observation prior to initiation of study drug). Note that samples from all participants were analyzed for each laboratory parameter. Values falling above or below the Roche predefined standard reference range were laboratory abnormalities labelled accordingly as 'high' or 'low'. Not every laboratory abnormality qualified as an adverse event; only if it was accompanied by clinical symptoms, resulted in a change in study treatment or in a medical intervention, or was clinically significant in the investigator's judgment. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase
Time frame: Baseline and Days 2, 4, 8, 11, 15, 29, 43, 57, 71, 85, and 113
Population: Safety analysis population. The number analyzed in the table below represents the number of participants without the specified laboratory abnormality at baseline. Samples from all participants were analyzed for each laboratory parameter.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Basophils, Absolute Count - High | 4 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | White Blood Cell Count - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Cholesterol - High | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Albumin - Low | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Alkaline Phosphatase - High | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | SGPT/ALT - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | SGOT/AST - Low | 5 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Blood Urea Nitrogen - Low | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Blood Urea Nitrogen - High | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Chloride - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Cholesterol - Low | 5 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Creatine Kinase - High | 3 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | C-Reactive Protein - High | 6 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Lactate Dehydrogenase - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Potassium - Low | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Sodium - Low | 6 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Bilirubin - High | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Protein, Total - Low | 3 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Triglycerides (Fasting) - High | 6 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Uric Acid - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Uric Acid - High | 4 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Fibrinogen - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Activated Partial Thromboplastin Time - Low | 13 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Eosinophils, Absolute Count - Low | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Eosinophils, Absolute Count - High | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Hematocrit - Low | 3 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Hemoglobin - High | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Lymphocytes, Absolute Count - Low | 4 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Lymphocytes, Absolute Count - High | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Erythrocyte Mean Corpuscular Hemoglobin (HGB)-High | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Erythro. Mean Corpuscular HGB Concentration - High | 8 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Monocytes, Absolute Count - High | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Neutrophils, Total, Absolute Count - Low | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Neutrophils, Total, Absolute Count - High | 2 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Platelet - High | 1 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Red Blood Cell Count - Low | 3 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Urine pH - High | 9 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Urine Specific Gravity - Low | 6 Participants |
| Emicizumab | Number of Participants With Laboratory Test Abnormalities | Urine Specific Gravity - High | 9 Participants |
Secondary
Time to Cmax (Tmax) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Population: The pharmacokinetics (PK) analysis population included all enrolled participants; participants were planned to be excluded if they deviated significantly from the protocol or if data was unavailable or incomplete which may influence the PK analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emicizumab | Time to Cmax (Tmax) of Emicizumab | 7.0 day |