Rare Diseases
Conditions
Brief summary
This is a randomized, placebo controlled and double-blinded study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD) and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects.
Detailed description
This is a randomized, placebo-controlled and double-blinded study to evaluate the PK, PD and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects, performed in Japan. The subjects, 8 in total, will be randomized to receive either emapalumab or matching placebo in a 3:1 ratio (emapalumab: placebo).
Interventions
DRUGNI-0501
emapalumab single i.v infusion (1 mg/kg)
DRUGSaline
Saline single i.v infusion
Sponsors
Swedish Orphan Biovitrum
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
SINGLE (Caregiver)
Masking description
Double blind
Eligibility
Sex/Gender
ALL
Age
20 Years to 50 Years
Healthy volunteers
Yes
Inclusion criteria
1. Healthy Japanese (male and female) subjects between 20 and 50 years (inclusive). 2. Body weight greater than 45 kg (female) or 50 kg (male) and a body mass index (BMI) \>18 kg/m2 and \< 30 kg/m2 (BMI= weight (kg) / height (m)²) 3. Vital signs in the following range: * Axillary body temperature: 35.2 - 37.5℃ * Heart rate (after at least 3 minutes of rest, measured in the supine position): 40-100 bpm * BP \< 140/80, mean of 3 readings after 15 minutes rest 4. Haemoglobin level equal or above 11 g/dL in females and 13 g/dL in males. 5. Subject having C-reactive protein (CRP) levels within the normal range (local laboratory range). 6. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant having agreed to use highly effective methods of contraception during dosing and for 6 months after receiving IMP. Highly effective contraception methods include: * Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. * Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment. * Male sterilization (at least 6 months prior to screening). For female patients on the study, the vasectomized male partner should be the sole partner for that patient, otherwise highly effective methods to be applied. * Use of oral (estrogen and progesterone) hormonal method of contraception, or placement of an intrauterine device (IUD) or intrauterine system (IUS) * In case of use of oral contraception women should have been stable on the same brand (or generic equivalent) for a minimum of 3 months before taking study treatment. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago. 7. Signed informed consent.
Exclusion criteria
1. Any clinically significant abnormality in the results of the safety laboratory tests. Subjects presenting a minor deviation from laboratory ranges could be enrolled if the investigator judge it to be non-clinically significant 2. Any clinically significant abnormality on the screening electrocardiogram (ECG), as judged by the investigator 3. History or clinical evidence of any disease and/or existence of any surgical or medical condition that might interfere with the absorption, distribution, metabolism or excretion of the study drugs 4. Actual presence or occurrence of any bacterial, viral, parasitic or fungal infection within the 4 weeks preceding IMP infusion 5. Positive results from serology examination for Hepatitis B surface antigen (HBsAg), Hepatitis C Virus (HCV), Human Immunodeficiency Virus (HIV), syphilis (TP-antigen and RPR) or pregnancy 6. Positive stool test for Shigella or salmonella infection. 7. Positive results from Sars-CoV-2 screening within 96 hours prior to randomization 8. History or clinical evidence suggestive of active or latent tuberculosis at screening. (i.e. test positive to the interferon gamma (IFNγ)-release assay) 9. History or presence of any severe allergic reactions 10. History of hypersensitivity or allergy to any component of emapalumab and/or valaciclovir hydrochloride 11. History or presence of any malignancy 12. History or presence of drug or alcohol abuse 13. Subject with a smoking history within the last 6 months prior to the time of screening 14. Immunization with a live vaccine within 6 weeks prior to receiving IMP and 12 weeks after IMP infusion 15. Experience of collected blood corresponding to any of the following * Component blood donation within 2 weeks before the screening test and within 2 weeks before the first study drug administration * Collection of 200 mL or more of blood (blood donation, etc.) from 4 weeks before the screening test until admission * Male subject who has experience of collection of 400 mL or more of blood (blood donation, etc.) from 12weeks before the screening test until admission. * Female subject who has experience of collection of 400 mL or more of blood (blood donation, etc.) from 16weeks before the screening test until admission. 16. Usage of any prescription drugs within 2 weeks or over-the-counter medication including herbal supplements (with the exception of multi-vitamins) within 1 week before IMP administration without prior approval from the investigator 17. Positive pregnancy test at screening or Day -1 18. Any circumstances or conditions, which, in the opinion of the investigator, may affect the subject's full participation in the study or compliance with the protocol 19. Enrollment in another concurrent clinical interventional study, or intake of another IMP, within four months or 5 half-lives (of the other IMP) prior to inclusion in this study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Maximum Observed Concentration of Emapalumab | Day 1 preinfusion, 1hr, 2hrs, 4hrs, 8hrs, 10hrs post dose, day 2, 3, 5, 8, week 2, 4, 6, 8, 10, 12, study completion week 14 or Withdrawal | The maximum observed concentration of emapalumab (Cmax) |
| The Time at Which the Maximum Concentration of Emapalumab is Observed | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | The time at which the maximum concentration of emapalumab is observed (Tmax) |
| Concentration of Emapalumab at End of Infusion | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | Concentration of emapalumab at end of infusion (CEnd of inf)) |
| Area Under the Plasma Concentration-time Curve | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | Area under the plasma concentration-time curve from emapalumab injection to time of last measurable concentration (AUClast) |
| Area Under the Concentration-time Curve Extrapolated to Infinity | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | Area under the plasma concentration-time curve from emapalumab injection extrapolated to infinity (AUCinf) |
| Emapalumab Elimination Half-life | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | Emapalumab elimination half-life (t1/2) |
| Apparent Total Body Clearance of Emapalumab From Plasma | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | Apparent total body clearance of emapalumab from plasma (CL) |
| Steady State Volume of Distribution | Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal | Apparent volume of distribution at steady state (Vss) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Levels of Albumin | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Albumin |
| Change in Levels of Prothrombin Time/International Normalized Ratio | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Prothrombin Time/International Normalized Ratio (PTINR) |
| Change in Levels of Fibrinogen | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Fibrinogen |
| Change in Levels of Complement C3 | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Complement C3 |
| Change in Levels of Creatinine | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Creatinine |
| Change in Levels of C-reactive Protein | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of C-reactive protein (CRP) |
| Change in Levels of Sodium | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Sodium |
| Change in Levels of Potassium | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Potassium |
| Change in Levels of Calcium | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Calcium |
| Change in Levels of Glucose | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Glucose |
| Change in Levels of HDL | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of HDL |
| Overall Summary of Adverse Events | Continuously from start of emapalumab infusion up to 14 weeks | Total number of reported adverse events |
| Change in Levels of BUN/Urea Haematology | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of BUN/Urea haematology |
| Change in Levels of Hemoglobin | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Hemoglobin |
| Change in Levels of Hematocrit | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Hematocrit |
| Change in Levels of Platelet Count | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Platelet count |
| Change in Levels of Neutrophils | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Neutrophils |
| Change in Levels of Red Blood Cells | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Red blood cells |
| Change in Levels of Immunoglobulin Levels | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Immunoglobulin levels (IgG) |
| Change in Levels of Coagulation Profile | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in Activated Partial Thromboplastin Clotting Time (APTT) |
| Presence of Anti-drug Antibodies and Neutralizing Antibodies | From Day 1 to Week 14 | Presence of anti-drug antibodies (ADA) and neutralizing antibodies (nAb) |
| Change in Levels of Complement C4 | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Complement C4 |
| Presence of Neutralizing Antibodies | From Day 1 to Week 14 | Presence of neutralizing antibodies (nAb) |
| Change in Levels of LDL | Baselilne, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of LDL |
| Change in Levels of Aspartate Aminotransferase | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Aspartate aminotransferase (AST) |
| Change in Levels of Alanine Aminotransferase | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Alanine aminotransferase (ALT) |
| Change in Levels of Direct Bilirubin | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Direct Bilirubin |
| Change in Levels of Total Bilirubin | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Total Bilirubin |
| Change in Levels of Uric Acid | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Uric acid |
| Change in Levels of Alkaline Phosphatase | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Alkaline phosphatase |
| Change in Levels of Total Protein | Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14) | Change from baseline in levels of Total protein |
Countries
Japan
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Emapalumab Emapalumab i.v infusion
NI-0501: Emapalumab single i.v infusion (1 mg/kg) | 6 |
| Placebo Saline i.v. infusion
Saline: Saline single i.v infusion | 2 |
| Total | 8 |
Baseline characteristics
| Characteristic | Placebo | Total | Emapalumab |
|---|---|---|---|
| Age, Continuous | 45 years | 40 years | 35.0 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 8 Participants | 6 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 2 Participants | 8 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 2 |
| other Total, other adverse events | 3 / 6 | 1 / 2 |
| serious Total, serious adverse events | 0 / 6 | 0 / 2 |
Outcome results
Primary
Apparent Total Body Clearance of Emapalumab From Plasma
Apparent total body clearance of emapalumab from plasma (CL)
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | Apparent Total Body Clearance of Emapalumab From Plasma | 0.006136 L/h |
Primary
Area Under the Concentration-time Curve Extrapolated to Infinity
Area under the plasma concentration-time curve from emapalumab injection extrapolated to infinity (AUCinf)
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | Area Under the Concentration-time Curve Extrapolated to Infinity | 12430000 ng*h/mL |
Primary
Area Under the Plasma Concentration-time Curve
Area under the plasma concentration-time curve from emapalumab injection to time of last measurable concentration (AUClast)
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | Area Under the Plasma Concentration-time Curve | 11690000 ng*h/mL |
Primary
Concentration of Emapalumab at End of Infusion
Concentration of emapalumab at end of infusion (CEnd of inf))
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | Concentration of Emapalumab at End of Infusion | 23230 ng/mL |
Primary
Emapalumab Elimination Half-life
Emapalumab elimination half-life (t1/2)
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | Emapalumab Elimination Half-life | 634.9 Hours |
Primary
Steady State Volume of Distribution
Apparent volume of distribution at steady state (Vss)
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | Steady State Volume of Distribution | 4.953 L |
Primary
The Maximum Observed Concentration of Emapalumab
The maximum observed concentration of emapalumab (Cmax)
Time frame: Day 1 preinfusion, 1hr, 2hrs, 4hrs, 8hrs, 10hrs post dose, day 2, 3, 5, 8, week 2, 4, 6, 8, 10, 12, study completion week 14 or Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | The Maximum Observed Concentration of Emapalumab | 23980 ng/mL |
Primary
The Time at Which the Maximum Concentration of Emapalumab is Observed
The time at which the maximum concentration of emapalumab is observed (Tmax)
Time frame: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Emapalumab | The Time at Which the Maximum Concentration of Emapalumab is Observed | 2.000 Hours |
Secondary
Change in Levels of Alanine Aminotransferase
Change from baseline in levels of Alanine aminotransferase (ALT)
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Alanine Aminotransferase | Day 3 | -2.0 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Week 4 | 1.0 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Day 5 | -0.5 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Day 1 | 0.5 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Day 8 | 4.5 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Week 2 | -3.0 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Week10 | 2.0 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Day 2 | 0.0 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Week 14 | 0.0 IU/L |
| Emapalumab | Change in Levels of Alanine Aminotransferase | Week 6 | -4.0 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Week 14 | 5.5 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Day 8 | 18.5 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Week 2 | 15.0 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Day 1 | 1.0 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Day 2 | 2.5 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Day 3 | 0.5 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Day 5 | 12.0 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Week 6 | 0.0 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Week10 | 8.5 IU/L |
| Placebo | Change in Levels of Alanine Aminotransferase | Week 4 | 19.5 IU/L |
Secondary
Change in Levels of Albumin
Change from baseline in levels of Albumin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Albumin | Day 1 | 0.10 g/dL |
| Emapalumab | Change in Levels of Albumin | Day 2 | 0.05 g/dL |
| Emapalumab | Change in Levels of Albumin | Day 3 | -0.15 g/dL |
| Emapalumab | Change in Levels of Albumin | Day 5 | 0.00 g/dL |
| Emapalumab | Change in Levels of Albumin | Day 8 | 0.30 g/dL |
| Emapalumab | Change in Levels of Albumin | Week 2 | -0.05 g/dL |
| Emapalumab | Change in Levels of Albumin | Week 4 | 0.10 g/dL |
| Emapalumab | Change in Levels of Albumin | Week 6 | -0.10 g/dL |
| Emapalumab | Change in Levels of Albumin | Week 10 | 0.30 g/dL |
| Emapalumab | Change in Levels of Albumin | Week 14 | 0.00 g/dL |
| Placebo | Change in Levels of Albumin | Week 6 | 0.00 g/dL |
| Placebo | Change in Levels of Albumin | Day 1 | 0.35 g/dL |
| Placebo | Change in Levels of Albumin | Week 2 | 0.00 g/dL |
| Placebo | Change in Levels of Albumin | Day 2 | 0.30 g/dL |
| Placebo | Change in Levels of Albumin | Week 14 | 0.25 g/dL |
| Placebo | Change in Levels of Albumin | Day 3 | 0.20 g/dL |
| Placebo | Change in Levels of Albumin | Week 4 | 0.10 g/dL |
| Placebo | Change in Levels of Albumin | Day 5 | 0.20 g/dL |
| Placebo | Change in Levels of Albumin | Week 10 | 0.05 g/dL |
| Placebo | Change in Levels of Albumin | Day 8 | 0.45 g/dL |
Secondary
Change in Levels of Alkaline Phosphatase
Change from baseline in levels of Alkaline phosphatase
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Alkaline Phosphatase | Day 1 | -34.0 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Day 2 | -26.5 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Day 3 | -23.5 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Day 5 | -28.0 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Day 8 | -6.5 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Week 2 | 9.5 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Week 4 | 4.0 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Week 6 | 8.0 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Week 10 | -0.5 U/L |
| Emapalumab | Change in Levels of Alkaline Phosphatase | Week 14 | -15.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Week 6 | 22.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Day 1 | 16.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Week 2 | 26.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Day 2 | 16.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Week 14 | 33.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Day 3 | 22.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Week 4 | 33.5 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Day 5 | 17.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Week 10 | 40.0 U/L |
| Placebo | Change in Levels of Alkaline Phosphatase | Day 8 | 35.0 U/L |
Secondary
Change in Levels of Aspartate Aminotransferase
Change from baseline in levels of Aspartate aminotransferase (AST)
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Day 1 | 0.5 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Day 2 | -0.5 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Day 3 | -2.5 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Day 5 | -1.5 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Day 8 | 1.0 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Week 2 | -2 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Week 4 | -0.5 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Week 6 | -2.0 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Week 10 | -1.5 IU/L |
| Emapalumab | Change in Levels of Aspartate Aminotransferase | Week 14 | -2.0 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Week 6 | 0.5 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Day 1 | 2.5 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Week 2 | 7.0 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Day 2 | 5.0 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Week 14 | 5.0 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Day 3 | 1.5 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Week 4 | 6.5 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Day 5 | 9.0 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Week 10 | 6.5 IU/L |
| Placebo | Change in Levels of Aspartate Aminotransferase | Day 8 | 11.5 IU/L |
Secondary
Change in Levels of BUN/Urea Haematology
Change from baseline in levels of BUN/Urea haematology
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of BUN/Urea Haematology | Day 3 | -1.05 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Week 2 | -1.90 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Week 4 | -1.40 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Day 2 | -0.55 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Week 6 | -2.20 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Day 5 | 0.80 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Week 10 | 0.95 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Day 1 | -1.35 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Week 14 | -1.70 mg/dL |
| Emapalumab | Change in Levels of BUN/Urea Haematology | Day 8 | -2.05 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Week 14 | 2.75 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Day 1 | -1.60 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Day 2 | 2.55 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Day 3 | 1.30 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Day 5 | 2.00 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Day 8 | -1.50 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Week 4 | 3.10 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Week 6 | -1.25 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Week 10 | -1.15 mg/dL |
| Placebo | Change in Levels of BUN/Urea Haematology | Week 2 | -2.30 mg/dL |
Secondary
Change in Levels of Calcium
Change from baseline in levels of Calcium
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Calcium | Week 4 | -0.15 mg/dL |
| Emapalumab | Change in Levels of Calcium | Week 14 | 0.00 mg/dL |
| Emapalumab | Change in Levels of Calcium | Week 2 | -0.05 mg/dL |
| Emapalumab | Change in Levels of Calcium | Day 1 | -0.05 mg/dL |
| Emapalumab | Change in Levels of Calcium | Week 6 | -0.40 mg/dL |
| Emapalumab | Change in Levels of Calcium | Day 3 | -0.10 mg/dL |
| Emapalumab | Change in Levels of Calcium | Day 2 | 0.00 mg/dL |
| Emapalumab | Change in Levels of Calcium | Day 5 | 0.05 mg/dL |
| Emapalumab | Change in Levels of Calcium | Week 10 | 0.30 mg/dL |
| Emapalumab | Change in Levels of Calcium | Day 8 | 0.15 mg/dL |
| Placebo | Change in Levels of Calcium | Week 10 | 0.00 mg/dL |
| Placebo | Change in Levels of Calcium | Day 8 | 0.10 mg/dL |
| Placebo | Change in Levels of Calcium | Week 2 | 0.10 mg/dL |
| Placebo | Change in Levels of Calcium | Week 4 | -0.05 mg/dL |
| Placebo | Change in Levels of Calcium | Week 6 | 0.15 mg/dL |
| Placebo | Change in Levels of Calcium | Day 1 | 0.40 mg/dL |
| Placebo | Change in Levels of Calcium | Week 14 | 0.55 mg/dL |
| Placebo | Change in Levels of Calcium | Day 2 | 0.15 mg/dL |
| Placebo | Change in Levels of Calcium | Day 3 | 0.30 mg/dL |
| Placebo | Change in Levels of Calcium | Day 5 | 0.20 mg/dL |
Secondary
Change in Levels of Coagulation Profile
Change from baseline in Activated Partial Thromboplastin Clotting Time (APTT)
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of patients analyzed are the number of available samples.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Coagulation Profile | Day 1 | 0.85 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Day 2 | -0.30 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Day 3 | 0.4 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Day 5 | -1.40 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Day 8 | 1.40 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Week 2 | -0.55 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Week 4 | -0.15 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Week 6 | -0.90 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Week 10 | 1.50 sec |
| Emapalumab | Change in Levels of Coagulation Profile | Week 14 | -1.80 sec |
| Placebo | Change in Levels of Coagulation Profile | Week 6 | -0.50 sec |
| Placebo | Change in Levels of Coagulation Profile | Day 1 | 0.4 sec |
| Placebo | Change in Levels of Coagulation Profile | Week 2 | 0.5 sec |
| Placebo | Change in Levels of Coagulation Profile | Day 2 | -0.65 sec |
| Placebo | Change in Levels of Coagulation Profile | Week 14 | -1.25 sec |
| Placebo | Change in Levels of Coagulation Profile | Day 3 | 1.80 sec |
| Placebo | Change in Levels of Coagulation Profile | Week 4 | -2.25 sec |
| Placebo | Change in Levels of Coagulation Profile | Day 5 | -0.80 sec |
| Placebo | Change in Levels of Coagulation Profile | Week 10 | -2.60 sec |
| Placebo | Change in Levels of Coagulation Profile | Day 8 | 0.30 sec |
Secondary
Change in Levels of Complement C3
Change from baseline in levels of Complement C3
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Complement C3 | Day 1 | 4.0 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Day 2 | 3.5 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Day 3 | 12.5 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Day 5 | 14.5 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Day 8 | 20.0 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Week 2 | 10.5 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Week 4 | 0.5 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Week 6 | -1.0 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Week 10 | 6.5 mg/dL |
| Emapalumab | Change in Levels of Complement C3 | Week 14 | -9.0 mg/dL |
| Placebo | Change in Levels of Complement C3 | Week 6 | -0.5 mg/dL |
| Placebo | Change in Levels of Complement C3 | Day 1 | 11.5 mg/dL |
| Placebo | Change in Levels of Complement C3 | Week 2 | 3.0 mg/dL |
| Placebo | Change in Levels of Complement C3 | Day 2 | 4.5 mg/dL |
| Placebo | Change in Levels of Complement C3 | Week 14 | 5.5 mg/dL |
| Placebo | Change in Levels of Complement C3 | Day 3 | 9.0 mg/dL |
| Placebo | Change in Levels of Complement C3 | Week 4 | 3.0 mg/dL |
| Placebo | Change in Levels of Complement C3 | Day 5 | 3.0 mg/dL |
| Placebo | Change in Levels of Complement C3 | Week 10 | 4.5 mg/dL |
| Placebo | Change in Levels of Complement C3 | Day 8 | 16.5 mg/dL |
Secondary
Change in Levels of Complement C4
Change from baseline in levels of Complement C4
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Complement C4 | Day 8 | 6.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Week 2 | 2.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Day 1 | 0.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Day 2 | 1.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Day 3 | 3.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Day 5 | 4.5 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Week 4 | 0.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Week 6 | -1.0 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Week 10 | -1.5 mg/dL |
| Emapalumab | Change in Levels of Complement C4 | Week 14 | -4.0 mg/dL |
| Placebo | Change in Levels of Complement C4 | Week 6 | 1.5 mg/dL |
| Placebo | Change in Levels of Complement C4 | Day 8 | 6.0 mg/dL |
| Placebo | Change in Levels of Complement C4 | Day 5 | 2.5 mg/dL |
| Placebo | Change in Levels of Complement C4 | Week 2 | 4.0 mg/dL |
| Placebo | Change in Levels of Complement C4 | Week 14 | 1.5 mg/dL |
| Placebo | Change in Levels of Complement C4 | Day 1 | 2.5 mg/dL |
| Placebo | Change in Levels of Complement C4 | Week 4 | -1.0 mg/dL |
| Placebo | Change in Levels of Complement C4 | Day 2 | 2.0 mg/dL |
| Placebo | Change in Levels of Complement C4 | Week 10 | -1.5 mg/dL |
| Placebo | Change in Levels of Complement C4 | Day 3 | 3.0 mg/dL |
Secondary
Change in Levels of C-reactive Protein
Change from baseline in levels of C-reactive protein (CRP)
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of C-reactive Protein | Week 2 | 0.010 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Day 3 | 0.645 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Day 5 | 0.155 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Week 4 | 0.000 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Week 6 | 0.030 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Week10 | 0.005 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Week 14 | -0.010 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Day 2 | 0.105 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Day 8 | 0.050 mg/dL |
| Emapalumab | Change in Levels of C-reactive Protein | Day 1 | 0.000 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Day 8 | -0.025 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Day 2 | 0.030 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Week10 | -0.025 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Day 3 | 0.025 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Day 5 | -0.035 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Week 2 | -0.050 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Week 14 | -0.025 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Week 4 | 0.065 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Day 1 | -0.015 mg/dL |
| Placebo | Change in Levels of C-reactive Protein | Week 6 | -0.060 mg/dL |
Secondary
Change in Levels of Creatinine
Change from baseline in levels of Creatinine
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Creatinine | Day 2 | 0.010 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Day 3 | 0.010 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Day 5 | 0.055 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Day 8 | -0.005 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Week 10 | -0.020 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Week 14 | 0.010 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Day 1 | 0.030 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Week 2 | -0.035 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Week 4 | -0.040 mg/dL |
| Emapalumab | Change in Levels of Creatinine | Week 6 | -0.030 mg/dL |
| Placebo | Change in Levels of Creatinine | Week 10 | -0.010 mg/dL |
| Placebo | Change in Levels of Creatinine | Day 2 | 0.020 mg/dL |
| Placebo | Change in Levels of Creatinine | Week 4 | 0.025 mg/dL |
| Placebo | Change in Levels of Creatinine | Day 3 | 0.025 mg/dL |
| Placebo | Change in Levels of Creatinine | Week 14 | 0.125 mg/dL |
| Placebo | Change in Levels of Creatinine | Day 5 | 0.035 mg/dL |
| Placebo | Change in Levels of Creatinine | Week 2 | -0.060 mg/dL |
| Placebo | Change in Levels of Creatinine | Day 8 | -0.005 mg/dL |
| Placebo | Change in Levels of Creatinine | Week 6 | 0.010 mg/dL |
| Placebo | Change in Levels of Creatinine | Day 1 | 0.095 mg/dL |
Secondary
Change in Levels of Direct Bilirubin
Change from baseline in levels of Direct Bilirubin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Direct Bilirubin | Day 1 | 0.15 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Day 5 | 0.05 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Week 14 | 0.00 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Day 8 | 0.10 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Day 2 | 0.15 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Week 2 | 0.00 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Week 10 | 0.05 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Week 4 | 0.05 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Day 3 | 0.10 mg/dL |
| Emapalumab | Change in Levels of Direct Bilirubin | Week 6 | 0.10 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Day 3 | -0.05 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Week 10 | -0.05 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Week 14 | -0.05 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Day 1 | 0.05 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Day 2 | 0.00 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Week 6 | 0.00 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Day 5 | -0.05 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Day 8 | 0.00 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Week 2 | -0.10 mg/dL |
| Placebo | Change in Levels of Direct Bilirubin | Week 4 | 0.00 mg/dL |
Secondary
Change in Levels of Fibrinogen
Change from baseline in levels of Fibrinogen
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Fibrinogen | Day 2 | 53.0 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Day 8 | 93.5 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Day 1 | 6.0 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Week 2 | -5.5 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Day 3 | 60.0 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Week 4 | -5.5 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Week 14 | -43.0 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Week 6 | -14.0 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Day 5 | 75.5 mg/dL |
| Emapalumab | Change in Levels of Fibrinogen | Week 10 | 10.5 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Week 14 | 7.5 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Week 10 | -5.0 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Day 1 | 49.5 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Day 2 | 103.5 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Day 3 | -97.0 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Day 5 | 6.0 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Day 8 | 84.0 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Week 2 | 2.5 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Week 4 | 2.5 mg/dL |
| Placebo | Change in Levels of Fibrinogen | Week 6 | 23.5 mg/dL |
Secondary
Change in Levels of Glucose
Change from baseline in levels of Glucose
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Glucose | Day 1 | -9.0 mg/dL |
| Emapalumab | Change in Levels of Glucose | Day 2 | -1.0 mg/dL |
| Emapalumab | Change in Levels of Glucose | Day 8 | -7.5 mg/dL |
| Emapalumab | Change in Levels of Glucose | Day 3 | 0.5 mg/dL |
| Emapalumab | Change in Levels of Glucose | Day 5 | -4.5 mg/dL |
| Emapalumab | Change in Levels of Glucose | Week 2 | -2.0 mg/dL |
| Emapalumab | Change in Levels of Glucose | Week 4 | -1.0 mg/dL |
| Emapalumab | Change in Levels of Glucose | Week 6 | 0.0 mg/dL |
| Emapalumab | Change in Levels of Glucose | Week 10 | 1.5 mg/dL |
| Emapalumab | Change in Levels of Glucose | Week 14 | 1.0 mg/dL |
| Placebo | Change in Levels of Glucose | Week 6 | 4.5 mg/dL |
| Placebo | Change in Levels of Glucose | Day 1 | -3.0 mg/dL |
| Placebo | Change in Levels of Glucose | Week 2 | 2.0 mg/dL |
| Placebo | Change in Levels of Glucose | Day 2 | 0.5 mg/dL |
| Placebo | Change in Levels of Glucose | Week 14 | 0.5 mg/dL |
| Placebo | Change in Levels of Glucose | Week 4 | 3.0 mg/dL |
| Placebo | Change in Levels of Glucose | Day 3 | 3.0 mg/dL |
| Placebo | Change in Levels of Glucose | Week 10 | 0.5 mg/dL |
| Placebo | Change in Levels of Glucose | Day 5 | -2.0 mg/dL |
| Placebo | Change in Levels of Glucose | Day 8 | 0.0 mg/dL |
Secondary
Change in Levels of HDL
Change from baseline in levels of HDL
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of HDL | Day 1 | -2.0 mg/dL |
| Emapalumab | Change in Levels of HDL | Day 2 | -3.5 mg/dL |
| Emapalumab | Change in Levels of HDL | Day 3 | -4.0 mg/dL |
| Emapalumab | Change in Levels of HDL | Day 5 | -8.0 mg/dL |
| Emapalumab | Change in Levels of HDL | Day 8 | -7.5 mg/dL |
| Emapalumab | Change in Levels of HDL | Week 2 | -7.0 mg/dL |
| Emapalumab | Change in Levels of HDL | Week 4 | -2.0 mg/dL |
| Emapalumab | Change in Levels of HDL | Week 6 | -5.0 mg/dL |
| Emapalumab | Change in Levels of HDL | Week 10 | 0.5 mg/dL |
| Emapalumab | Change in Levels of HDL | Week 14 | -5.0 mg/dL |
| Placebo | Change in Levels of HDL | Week 6 | -1.0 mg/dL |
| Placebo | Change in Levels of HDL | Day 1 | 0.0 mg/dL |
| Placebo | Change in Levels of HDL | Week 2 | 0.0 mg/dL |
| Placebo | Change in Levels of HDL | Day 2 | -1.0 mg/dL |
| Placebo | Change in Levels of HDL | Week 14 | 0.5 mg/dL |
| Placebo | Change in Levels of HDL | Day 3 | 1.0 mg/dL |
| Placebo | Change in Levels of HDL | Week 4 | -2.0 mg/dL |
| Placebo | Change in Levels of HDL | Day 5 | -1.5 mg/dL |
| Placebo | Change in Levels of HDL | Week 10 | -0.5 mg/dL |
| Placebo | Change in Levels of HDL | Day 8 | 2.5 mg/dL |
Secondary
Change in Levels of Hematocrit
Change from baseline in levels of Hematocrit
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Hematocrit | Week 10 | 3.05 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Day 2 | 1.10 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Week 6 | 1.00 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Day 3 | 0.70 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Week 14 | 0.1 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Day 5 | 0.90 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Week 4 | 0.15 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Day 8 | 2.70 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Day 1 | 1.6 % of red blood cells in blood |
| Emapalumab | Change in Levels of Hematocrit | Week 2 | -0.40 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Day 1 | 2.80 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Week 4 | 0.95 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Week 6 | 0.85 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Week 10 | 2.70 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Week 14 | 2.45 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Week 2 | 0.3 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Day 2 | 2.10 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Day 3 | 1.55 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Day 5 | 0.7 % of red blood cells in blood |
| Placebo | Change in Levels of Hematocrit | Day 8 | 2.15 % of red blood cells in blood |
Secondary
Change in Levels of Hemoglobin
Change from baseline in levels of Hemoglobin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Hemoglobin | Day 1 | 0.4 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Day 2 | 0.35 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Day 3 | 0.20 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Day 5 | 0.25 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Day 8 | 0.75 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Week 2 | -0.25 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Week 4 | 0.00 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Week 6 | -0.20 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Week 10 | 0.90 g/dL |
| Emapalumab | Change in Levels of Hemoglobin | Week 14 | 0.20 g/dL |
| Placebo | Change in Levels of Hemoglobin | Week 6 | 0.05 g/dL |
| Placebo | Change in Levels of Hemoglobin | Day 1 | 0.75 g/dL |
| Placebo | Change in Levels of Hemoglobin | Week 2 | 0.10 g/dL |
| Placebo | Change in Levels of Hemoglobin | Day 2 | 0.55 g/dL |
| Placebo | Change in Levels of Hemoglobin | Week 14 | 1.25 g/dL |
| Placebo | Change in Levels of Hemoglobin | Day 3 | 0.45 g/dL |
| Placebo | Change in Levels of Hemoglobin | Week 4 | 0.20 g/dL |
| Placebo | Change in Levels of Hemoglobin | Day 5 | 0.25 g/dL |
| Placebo | Change in Levels of Hemoglobin | Week 10 | 0.65 g/dL |
| Placebo | Change in Levels of Hemoglobin | Day 8 | 0.7 g/dL |
Secondary
Change in Levels of Immunoglobulin Levels
Change from baseline in levels of Immunoglobulin levels (IgG)
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Immunoglobulin Levels | Day 1 | 69.5 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Day 2 | 28.5 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Day 3 | 5.0 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Day 5 | 6.0 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Day 8 | 125.5 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Week 2 | 14.5 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Week 4 | 29.5 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Week 6 | 2.0 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Week 10 | 98.0 mg/dL |
| Emapalumab | Change in Levels of Immunoglobulin Levels | Week 14 | 1.0 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Week 6 | 36.0 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Day 1 | 119.5 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Week 2 | 48.0 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Day 2 | 72.0 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Week 14 | 89.5 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Day 3 | 78 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Week 4 | 34.5 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Day 5 | 16.0 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Week 10 | 49.5 mg/dL |
| Placebo | Change in Levels of Immunoglobulin Levels | Day 8 | 156.5 mg/dL |
Secondary
Change in Levels of LDL
Change from baseline in levels of LDL
Time frame: Baselilne, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of LDL | Day 1 | 11.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Day 2 | 5.5 mg/dL |
| Emapalumab | Change in Levels of LDL | Day 3 | 3.5 mg/dL |
| Emapalumab | Change in Levels of LDL | Day 5 | 3.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Day 8 | 7.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Week 2 | -7.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Week 4 | 2.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Week 6 | 3.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Week 10 | 9.0 mg/dL |
| Emapalumab | Change in Levels of LDL | Week 14 | -8.0 mg/dL |
| Placebo | Change in Levels of LDL | Week 6 | 3.5 mg/dL |
| Placebo | Change in Levels of LDL | Day 1 | 16.5 mg/dL |
| Placebo | Change in Levels of LDL | Week 2 | 17.0 mg/dL |
| Placebo | Change in Levels of LDL | Day 2 | 17.0 mg/dL |
| Placebo | Change in Levels of LDL | Week 14 | 19.5 mg/dL |
| Placebo | Change in Levels of LDL | Day 3 | 19.5 mg/dL |
| Placebo | Change in Levels of LDL | Week 4 | 0.0 mg/dL |
| Placebo | Change in Levels of LDL | Day 5 | 7.5 mg/dL |
| Placebo | Change in Levels of LDL | Week 10 | 3.5 mg/dL |
| Placebo | Change in Levels of LDL | Day 8 | 13.0 mg/dL |
Secondary
Change in Levels of Neutrophils
Change from baseline in levels of Neutrophils
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Neutrophils | Week 14 | -5.40 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Day 1 | -0.30 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Day 2 | 17.45 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Day 3 | 9.70 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Day 5 | 0.75 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Day 8 | 1.65 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Week 2 | 2.50 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Week 4 | -1.05 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Week 6 | -4.10 % of white blood cells |
| Emapalumab | Change in Levels of Neutrophils | Week 10 | 1.45 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Week 4 | 2.10 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Week 14 | 1.65 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Day 8 | 1.30 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Day 1 | 0.35 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Week 10 | 0.95 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Day 2 | -0.40 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Week 2 | 5.10 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Day 3 | -0.85 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Week 6 | 0.55 % of white blood cells |
| Placebo | Change in Levels of Neutrophils | Day 5 | -16.25 % of white blood cells |
Secondary
Change in Levels of Platelet Count
Change from baseline in levels of Platelet count
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Platelet Count | Day 1 | 0.40 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Day 2 | -0.15 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Day 3 | -0.1 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Day 5 | 0.05 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Day 8 | 1.8 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Week 2 | 0.70 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Week 4 | 0.05 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Week 6 | 0.4 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Week 10 | 0.5 x10^10/L |
| Emapalumab | Change in Levels of Platelet Count | Week 14 | -0.10 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Week 6 | 3.10 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Day 1 | 1.20 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Week 2 | 2.30 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Day 2 | 1.10 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Week 14 | 4.65 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Day 3 | 1.40 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Week 4 | 1.55 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Day 5 | 1.8 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Week 10 | 2.40 x10^10/L |
| Placebo | Change in Levels of Platelet Count | Day 8 | 3.70 x10^10/L |
Secondary
Change in Levels of Potassium
Change from baseline in levels of Potassium
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Potassium | Week 10 | 0.05 mEq/L |
| Emapalumab | Change in Levels of Potassium | Week 14 | 0.00 mEq/L |
| Emapalumab | Change in Levels of Potassium | Day 1 | 0.00 mEq/L |
| Emapalumab | Change in Levels of Potassium | Day 2 | 0.00 mEq/L |
| Emapalumab | Change in Levels of Potassium | Day 3 | 0.15 mEq/L |
| Emapalumab | Change in Levels of Potassium | Day 5 | 0.15 mEq/L |
| Emapalumab | Change in Levels of Potassium | Day 8 | -0.15 mEq/L |
| Emapalumab | Change in Levels of Potassium | Week 2 | 0.15 mEq/L |
| Emapalumab | Change in Levels of Potassium | Week 4 | 0.00 mEq/L |
| Emapalumab | Change in Levels of Potassium | Week 6 | 0.00 mEq/L |
| Placebo | Change in Levels of Potassium | Week 2 | 0.10 mEq/L |
| Placebo | Change in Levels of Potassium | Week 10 | -0.20 mEq/L |
| Placebo | Change in Levels of Potassium | Day 5 | 0.00 mEq/L |
| Placebo | Change in Levels of Potassium | Week 14 | 0.30 mEq/L |
| Placebo | Change in Levels of Potassium | Week 6 | 0.40 mEq/L |
| Placebo | Change in Levels of Potassium | Day 1 | 0.60 mEq/L |
| Placebo | Change in Levels of Potassium | Day 8 | -0.25 mEq/L |
| Placebo | Change in Levels of Potassium | Day 2 | 0.15 mEq/L |
| Placebo | Change in Levels of Potassium | Week 4 | 0.35 mEq/L |
| Placebo | Change in Levels of Potassium | Day 3 | 0.40 mEq/L |
Secondary
Change in Levels of Prothrombin Time/International Normalized Ratio
Change from baseline in levels of Prothrombin Time/International Normalized Ratio (PTINR)
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 1 | 0.065 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 2 | 0.045 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 3 | 0.075 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 5 | 0.015 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 8 | -0.005 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 2 | -0.020 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 4 | -0.040 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 6 | -0.050 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 10 | -0.030 Unitless |
| Emapalumab | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 14 | -0.030 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 6 | -0.050 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 1 | -0.005 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 2 | -0.060 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 2 | -0.035 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 14 | -0.085 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 3 | 0.010 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 4 | -0.055 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 5 | -0.025 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Week 10 | -0.065 Unitless |
| Placebo | Change in Levels of Prothrombin Time/International Normalized Ratio | Day 8 | -0.055 Unitless |
Secondary
Change in Levels of Red Blood Cells
Change from baseline in levels of Red blood cells
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Red Blood Cells | Day 1 | 18 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Day 2 | 13.5 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Day 3 | 9.0 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Day 5 | 12.0 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Day 8 | 29.5 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Week 2 | -4.0 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Week 4 | -1.0 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Week 6 | -6.0 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Week 10 | 23.0 cellsx10^10/L |
| Emapalumab | Change in Levels of Red Blood Cells | Week 14 | -6.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Week 6 | -0.5 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Day 1 | 27.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Week 2 | 0.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Day 2 | 20.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Week 14 | 18.5 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Day 3 | 15.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Week 4 | 2.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Day 5 | 8.5 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Week 10 | 20.0 cellsx10^10/L |
| Placebo | Change in Levels of Red Blood Cells | Day 8 | 23.5 cellsx10^10/L |
Secondary
Change in Levels of Sodium
Change from baseline in levels of Sodium
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Sodium | Day 1 | -1.0 mEq/L |
| Emapalumab | Change in Levels of Sodium | Day 2 | -0.5 mEq/L |
| Emapalumab | Change in Levels of Sodium | Day 3 | -1.5 mEq/L |
| Emapalumab | Change in Levels of Sodium | Day 5 | 0.00 mEq/L |
| Emapalumab | Change in Levels of Sodium | Day 8 | 0.0 mEq/L |
| Emapalumab | Change in Levels of Sodium | Week 2 | -0.5 mEq/L |
| Emapalumab | Change in Levels of Sodium | Week 4 | -0.5 mEq/L |
| Emapalumab | Change in Levels of Sodium | Week 6 | 0.0 mEq/L |
| Emapalumab | Change in Levels of Sodium | Week 10 | -1.5 mEq/L |
| Emapalumab | Change in Levels of Sodium | Week 14 | -1.0 mEq/L |
| Placebo | Change in Levels of Sodium | Week 6 | 5.0 mEq/L |
| Placebo | Change in Levels of Sodium | Day 1 | 3.5 mEq/L |
| Placebo | Change in Levels of Sodium | Week 2 | 1.0 mEq/L |
| Placebo | Change in Levels of Sodium | Day 2 | 1.5 mEq/L |
| Placebo | Change in Levels of Sodium | Week 14 | 3.5 mEq/L |
| Placebo | Change in Levels of Sodium | Day 3 | 2.0 mEq/L |
| Placebo | Change in Levels of Sodium | Week 4 | 3.5 mEq/L |
| Placebo | Change in Levels of Sodium | Day 5 | 2.5 mEq/L |
| Placebo | Change in Levels of Sodium | Week 10 | 0.5 mEq/L |
| Placebo | Change in Levels of Sodium | Day 8 | 2.0 mEq/L |
Secondary
Change in Levels of Total Bilirubin
Change from baseline in levels of Total Bilirubin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Total Bilirubin | Day 1 | 0.35 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Day 2 | 0.55 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Day 3 | 0.15 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Day 5 | 0.10 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Day 8 | 0.20 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Week 2 | -0.05 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Week 4 | 0.15 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Week 6 | 0.10 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Week 10 | 0.15 mg/dL |
| Emapalumab | Change in Levels of Total Bilirubin | Week 14 | 0.10 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Week 6 | 0.05 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Day 1 | 0.30 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Week 2 | -0.20 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Day 2 | 0.10 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Week 14 | -0.15 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Day 3 | -0.10 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Week 4 | -0.05 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Day 5 | -0.20 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Week 10 | 0.00 mg/dL |
| Placebo | Change in Levels of Total Bilirubin | Day 8 | 0.05 mg/dL |
Secondary
Change in Levels of Total Protein
Change from baseline in levels of Total protein
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Total Protein | Day 3 | 0.20 g/dL |
| Emapalumab | Change in Levels of Total Protein | Week 2 | -0.05 g/dL |
| Emapalumab | Change in Levels of Total Protein | Day 2 | 0.25 g/dL |
| Emapalumab | Change in Levels of Total Protein | Week 4 | 0.05 g/dL |
| Emapalumab | Change in Levels of Total Protein | Day 5 | 0.15 g/dL |
| Emapalumab | Change in Levels of Total Protein | Week 6 | -0.10 g/dL |
| Emapalumab | Change in Levels of Total Protein | Day 1 | 0.25 g/dL |
| Emapalumab | Change in Levels of Total Protein | Week 10 | 0.4 g/dL |
| Emapalumab | Change in Levels of Total Protein | Day 8 | 0.50 g/dL |
| Emapalumab | Change in Levels of Total Protein | Week 14 | 0.10 g/dL |
| Placebo | Change in Levels of Total Protein | Day 8 | 0.65 g/dL |
| Placebo | Change in Levels of Total Protein | Day 1 | 0.30 g/dL |
| Placebo | Change in Levels of Total Protein | Day 2 | 0.25 g/dL |
| Placebo | Change in Levels of Total Protein | Day 3 | 0.35 g/dL |
| Placebo | Change in Levels of Total Protein | Day 5 | 0.00 g/dL |
| Placebo | Change in Levels of Total Protein | Week 14 | 0.55 g/dL |
| Placebo | Change in Levels of Total Protein | Week 2 | 0.10 g/dL |
| Placebo | Change in Levels of Total Protein | Week 4 | -0.10 g/dL |
| Placebo | Change in Levels of Total Protein | Week 6 | 0.05 g/dL |
| Placebo | Change in Levels of Total Protein | Week 10 | 0.15 g/dL |
Secondary
Change in Levels of Uric Acid
Change from baseline in levels of Uric acid
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Emapalumab | Change in Levels of Uric Acid | Day 1 | 0.15 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Day 2 | 0.40 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Day 3 | 0.40 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Day 5 | 0.35 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Day 8 | 0.25 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Week 2 | -0.10 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Week 4 | 0.25 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Week 6 | 0.00 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Week 10 | 0.40 mg/dL |
| Emapalumab | Change in Levels of Uric Acid | Week 14 | 0.10 mg/dL |
| Placebo | Change in Levels of Uric Acid | Week 6 | 0.10 mg/dL |
| Placebo | Change in Levels of Uric Acid | Day 1 | 0.10 mg/dL |
| Placebo | Change in Levels of Uric Acid | Week 2 | -0.40 mg/dL |
| Placebo | Change in Levels of Uric Acid | Day 2 | 0.15 mg/dL |
| Placebo | Change in Levels of Uric Acid | Week 14 | 0.85 mg/dL |
| Placebo | Change in Levels of Uric Acid | Day 3 | 0.15 mg/dL |
| Placebo | Change in Levels of Uric Acid | Week 4 | -0.05 mg/dL |
| Placebo | Change in Levels of Uric Acid | Day 5 | 0.20 mg/dL |
| Placebo | Change in Levels of Uric Acid | Week 10 | 0.55 mg/dL |
| Placebo | Change in Levels of Uric Acid | Day 8 | 0.20 mg/dL |
Secondary
Overall Summary of Adverse Events
Total number of reported adverse events
Time frame: Continuously from start of emapalumab infusion up to 14 weeks
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Emapalumab | Overall Summary of Adverse Events | Serious TEAEs | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | ADRs leading to dose reduced | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | TEAEs leading to drug withdrawn | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | TEAEs leading to drug interrupted | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | Adverse Drug Reactions (ADRs) | 3 number of events |
| Emapalumab | Overall Summary of Adverse Events | ADRs leading to drug interrupted | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | ADRs leading to drug withdrawn | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | TEAEs leading to death | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | Serious ADRs | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | ADRs leading to death | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | TEAEs leading to dose reduced | 0 number of events |
| Emapalumab | Overall Summary of Adverse Events | Infusion Related Reactions, (IRRs) | 2 number of events |
| Emapalumab | Overall Summary of Adverse Events | All Treatment emergent adverse events (TEAEs) | 3 number of events |
| Placebo | Overall Summary of Adverse Events | Infusion Related Reactions, (IRRs) | 0 number of events |
| Placebo | Overall Summary of Adverse Events | All Treatment emergent adverse events (TEAEs) | 1 number of events |
| Placebo | Overall Summary of Adverse Events | Adverse Drug Reactions (ADRs) | 0 number of events |
| Placebo | Overall Summary of Adverse Events | Serious TEAEs | 0 number of events |
| Placebo | Overall Summary of Adverse Events | Serious ADRs | 0 number of events |
| Placebo | Overall Summary of Adverse Events | TEAEs leading to drug withdrawn | 0 number of events |
| Placebo | Overall Summary of Adverse Events | ADRs leading to drug withdrawn | 0 number of events |
| Placebo | Overall Summary of Adverse Events | TEAEs leading to dose reduced | 0 number of events |
| Placebo | Overall Summary of Adverse Events | ADRs leading to dose reduced | 0 number of events |
| Placebo | Overall Summary of Adverse Events | TEAEs leading to drug interrupted | 0 number of events |
| Placebo | Overall Summary of Adverse Events | ADRs leading to drug interrupted | 0 number of events |
| Placebo | Overall Summary of Adverse Events | TEAEs leading to death | 0 number of events |
| Placebo | Overall Summary of Adverse Events | ADRs leading to death | 0 number of events |
Secondary
Presence of Anti-drug Antibodies and Neutralizing Antibodies
Presence of anti-drug antibodies (ADA) and neutralizing antibodies (nAb)
Time frame: From Day 1 to Week 14
Population: Number of analyzed are the number of available samples
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Emapalumab | Presence of Anti-drug Antibodies and Neutralizing Antibodies | Baseline | 0 Participants |
| Emapalumab | Presence of Anti-drug Antibodies and Neutralizing Antibodies | Week 14 | 0 Participants |
Secondary
Presence of Neutralizing Antibodies
Presence of neutralizing antibodies (nAb)
Time frame: From Day 1 to Week 14
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Emapalumab | Presence of Neutralizing Antibodies | NA Participants |