Genetic Disease, Kidney Diseases, Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Urologic Diseases
Conditions
Keywords
PH1, PH2, Primary Hyperoxaluria, RNAi, RNAi therapeutic, GalNAc, LDHA gene, LDH, siRNA
Brief summary
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
Interventions
DRUGDCR-PHXC
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
DRUGSterile Normal Saline (0.9% NaCl)
Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo
Sponsors
Novo Nordisk A/S
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Capable and willing to provide written informed consent or assent * Documented diagnosis of PH1 or PH2, confirmed by genotyping * Must meet the 24 hour urine oxalate excretion requirements * Less than 20% variation between the two 24-hour urinary creatinine excretion values derived from the two 24-hour urine collections in the screening period * Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA Key
Exclusion criteria
* Renal or hepatic transplantation (prior or planned within the study period) * Currently on dialysis or anticipated requirement for dialysis during the study period * Plasma oxalate \>30 µmol/L * Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations) * Use of an RNA interference (RNAi) drug within the last 6 months * Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening * Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \>1.5 times upper limit of normal (ULN) for age and gender * Inability or unwillingness to comply with study procedures
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | From Day 90 to 180 | The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | Baseline, Day 180 | Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm\^2) of kidney stones is presented. |
| Percent Change From Baseline to Day 180 in the Number of Kidney Stones | Baseline, Day 180 | Percent change from baseline to Day 180 in the number of kidney stones is presented. |
| Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | Baseline, Day 180 | Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented. |
| Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180 | Baseline, Day 180 | Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation. |
| Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | From Baseline up to Day 180 | Number of TEAEs and TESAEs are presented. An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE was defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalisation, results in persistent disability/incapacity or is a congenital anomaly/birth defect. TEAE was defined as any AE with an onset date/time on or after administration (including any partial administration) of the first dose of study intervention and through the study completion date from the end of study case report form (CRF). |
| Change From Baseline in Electrocardiogram (ECG): Heart Rate | Baseline, Day 180 | Change from baseline in heart rate is presented. |
| Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | Baseline, Day 180 | Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented. |
| Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Baseline up to Day 180 | Number of participants who had most abnormal post-baseline shift in physical examination are presented. Physical examination shifts were categories into 4 categories: 1) missing; 2) normal; 3) abnormal-not clinically significant (NCS) and 4) abnormal-clinically significant (CS). Each category was presented according body systems including: 1) eyes, ears, nose and throat; 2) chest/respiratory; 3) heart/cardiovascular; 4) gastrointestinal/liver; 5) musculoskeletal/extremities; 6) dermatological/skin; 7) thyroid/neck; 8) lymph nodes; 9) neurological. |
| Change From Baseline in Vital Signs: Height | Baseline, Day 180 | Change from baseline to Day 180 in height is presented. |
| Change From Baseline in Vital Signs: Weight | Baseline, Day 180 | Change from baseline to Day 180 in weight is presented. |
| Change From Baseline in Vital Signs: Body Mass Index (BMI) | Baseline, Day 180 | Change from baseline to Day 180 in BMI is presented. |
| Change From Baseline in Vital Signs: Oral Body Temperature | Baseline, Day 180 | Change from baseline to Day 180 in oral body temperature is presented. |
| Change From Baseline in Vital Signs: Heart Rate | Baseline, Day 180 | Change from baseline to Day 180 in heart rate is presented. |
| Change From Baseline in Vital Signs: Respiratory Rate | Baseline, Day 180 | Change from baseline to Day 180 in respiratory rate is presented. |
| Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure | Baseline, Day 180 | Change from baseline to Day 180 in systolic and diastolic blood pressure is presented. |
| Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Baseline, Day 180 | Change from baseline to Day 180 in alanine aminotransferase, aspartate aminotransferase, glutamate dehydrogenase, gamma glutamyl transferase, alkaline phosphatase, lactate dehydrogenase and creatine kinase are presented. |
| Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Baseline, Day 180 | Change from baseline to Day 180 in bilirubin, direct bilirubin and creatinine are presented. |
| Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | From Day 90 to 180 | Percentage of participants whose 24-hour Uox values normalized or near-normalized on at least 2 consecutive visits are presented. Normalization of Uox was defined as less than (\<) 0.46 millimole per 24 hours (mmol/24 hours) and near normalization was defined as greater than or equal to (\>=) 0.46 to \< 0.60 mmol/24 hours (values adjusted per 1.73 square meter \[1.73 m\^2\] body surface area \[BSA\] in participants aged \<18 years). |
| Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Baseline, Day 180 | Change from baseline to Day 180 in sodium, chloride, potassium and urea are presented. |
| Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6 | Baseline, Day 180 | Change from baseline to Day 180 in vitamin B6 is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes | Baseline, Day 180 | Change from baseline to Day 180 in erythrocytes is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration | Baseline, Day 180 | Change from baseline to Day 180 in hemoglobin and erythrocytes mean corpuscular hemoglobin concentration are presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit | Baseline, Day 180 | Change from baseline to Day 180 in hematocrit is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume | Baseline, Day 180 | Change from baseline to Day 180 in ery. mean corpuscular volume and mean platelet volume are presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin | Baseline, Day 180 | Change from baseline to Day 180 in erythrocytes mean corpuscular hemoglobin is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Baseline, Day 180 | Change from baseline to Day 180 in reticulocytes, platelets, leukocytes, lymphocytes, monocytes, eosinophils, basophils, neutrophils are presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes | Baseline, Day 180 | Change from baseline to Day 180 in lymphocytes/leukocytes is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes | Baseline, Day 180 | Change from baseline to Day 180 in monocytes/leukocytes is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte | Baseline, Day 180 | Change from baseline to Day 180 in eosinophils/leukocytes is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes | Baseline, Day 180 | Change from baseline to Day 180 in basophils/leukocytes is presented. |
| Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes | Baseline, Day 180 | Change from baseline to Day 180 in neutrophils/leukocytes is presented. |
| Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity | Baseline, Day 180 | Change from baseline to Day 180 in urine specific gravity is presented. |
| Change From Baseline in Clinical Urinalysis Laboratory Tests: pH | Baseline, Day 180 | Change from baseline to Day 180 in urine pH is presented. |
| Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC | For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours (hrs) postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose | The Cmax was defined as the maximum observed plasma concentration during a dosing interval. Data for this endpoint is reported only for adults and adolescent participants from PK population. |
| Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC | For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose | AUC0-last was defined as the area under the curve from time of administration to the last measurable concentration. Data for this endpoint is reported only for adults and adolescent participants from PK population. |
| Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin | Baseline, Day 180 | Change from baseline to Day 180 in protein and albumin are presented. |
Countries
Australia, Canada, France, Germany, Israel, Italy, Japan, Lebanon, Netherlands, New Zealand, Poland, Romania, Spain, United Kingdom, United States
Participant flow
Recruitment details
The study was conducted at 19 sites in France, Spain, Italy, Netherlands, Germany, United Kingdom, Australia, United States, Canada, Lebanon, and Japan.
Pre-assignment details
A total of 57 participants were screened, of which 35 participants were randomized: 23 to DCR-PHXC and 12 to placebo. All 35 randomized participants received at least one dose of study drug.
Participants by arm
| Arm | Count |
|---|---|
| DCR-PHXC Participants aged \>=12 years weighing \>=50 kg received nedosiran 170 mg (160 mg/mL FAE); participants \>=12 years weighing \<50 kg received 136 mg (128 mg FAE); and participants 6-11 years received 3.5 mg/kg (3.3 mg/kg FAE) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150. | 23 |
| Placebo Participants received the equivalent volume of nedosiran matching placebo SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150. | 12 |
| Total | 35 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 1 | 1 |
Baseline characteristics
| Characteristic | DCR-PHXC | Placebo | Total |
|---|---|---|---|
| Age, Continuous | 23.7 years STANDARD_DEVIATION 11.95 | 23.6 years STANDARD_DEVIATION 11.48 | 23.7 years STANDARD_DEVIATION 11.62 |
| Age, Customized Between 12 to 17 years | 6 Participants | 4 Participants | 10 Participants |
| Age, Customized Between 18 to 64 years | 14 Participants | 6 Participants | 20 Participants |
| Age, Customized Between 2 to 11 years | 3 Participants | 2 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 0 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 19 Participants | 11 Participants | 30 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 2 Participants | 1 Participants | 3 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 6 Participants | 0 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 | 1 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants | 1 Participants | 3 Participants |
| Race (NIH/OMB) White | 15 Participants | 10 Participants | 25 Participants |
| Sex: Female, Male Female | 12 Participants | 6 Participants | 18 Participants |
| Sex: Female, Male Male | 11 Participants | 6 Participants | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 23 | 0 / 12 |
| other Total, other adverse events | 16 / 23 | 10 / 12 |
| serious Total, serious adverse events | 1 / 23 | 2 / 12 |
Outcome results
Primary
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC.
Time frame: From Day 90 to 180
Population: MITT population included all participants in the ITT population who had at least one efficacy assessment after the Day 90 dosing visit, where the ITT population included all participants who were randomized and had at least one post-baseline efficacy assessment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | 3507.4 Percent change in 24-hour Uox AUC | Standard Error 788.49 |
| Placebo | AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | -1664.4 Percent change in 24-hour Uox AUC | Standard Error 1189.96 |
p-value: <0.000195% CI: [2929.3, 7414.2]ANCOVA
Secondary
Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC
AUC0-last was defined as the area under the curve from time of administration to the last measurable concentration. Data for this endpoint is reported only for adults and adolescent participants from PK population.
Time frame: For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
Population: The PK population included all participants in the safety population without major dosing violations, where safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data and number analysed signifies participants with available data for each specified category.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC | Day 1 | 12500 hours*nanograms per millilitre (h*ng/mL) | Geometric Coefficient of Variation 29.3 |
| DCR-PHXC | Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC | Day 30 | 12800 hours*nanograms per millilitre (h*ng/mL) | Geometric Coefficient of Variation 38.4 |
| DCR-PHXC | Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC | Day 150 | 6100 hours*nanograms per millilitre (h*ng/mL) | Geometric Coefficient of Variation 46.2 |
| Placebo | Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC | Day 1 | 6450 hours*nanograms per millilitre (h*ng/mL) | Geometric Coefficient of Variation 54.5 |
| Placebo | Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC | Day 30 | 6400 hours*nanograms per millilitre (h*ng/mL) | Geometric Coefficient of Variation 45.1 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase
Change from baseline to Day 180 in alanine aminotransferase, aspartate aminotransferase, glutamate dehydrogenase, gamma glutamyl transferase, alkaline phosphatase, lactate dehydrogenase and creatine kinase are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data and number analysed signifies participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Glutamate dehydrogenase | -0.15 Units per litre (U/L) | Standard Deviation 1.913 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Alkaline phosphatase | 3.0 Units per litre (U/L) | Standard Deviation 29.11 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Aspartate aminotransferase | -0.2 Units per litre (U/L) | Standard Deviation 4.95 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Lactate dehydrogenase | 4.1 Units per litre (U/L) | Standard Deviation 20.51 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Gamma glutamyl transferase | 2.7 Units per litre (U/L) | Standard Deviation 4.12 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Creatine kinase | 7.5 Units per litre (U/L) | Standard Deviation 59.28 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Alanine aminotransferase | 1.0 Units per litre (U/L) | Standard Deviation 8.59 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Creatine kinase | -34.1 Units per litre (U/L) | Standard Deviation 160.46 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Alanine aminotransferase | -1.1 Units per litre (U/L) | Standard Deviation 4.59 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Aspartate aminotransferase | -1.3 Units per litre (U/L) | Standard Deviation 4.73 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Glutamate dehydrogenase | -0.38 Units per litre (U/L) | Standard Deviation 1.064 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Gamma glutamyl transferase | -0.2 Units per litre (U/L) | Standard Deviation 3.03 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Alkaline phosphatase | -16.8 Units per litre (U/L) | Standard Deviation 41.78 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase | Lactate dehydrogenase | 1.0 Units per litre (U/L) | Standard Deviation 20.72 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine
Change from baseline to Day 180 in bilirubin, direct bilirubin and creatinine are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data and number analysed signifies participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Bilirubin | 1.0 micromoles per litre (umol/L) | Standard Deviation 3.18 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Direct bilirubin | 0.1 micromoles per litre (umol/L) | Standard Deviation 1.32 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Creatinine | -1.2 micromoles per litre (umol/L) | Standard Deviation 10.81 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Bilirubin | 0.4 micromoles per litre (umol/L) | Standard Deviation 1.8 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Direct bilirubin | 0.1 micromoles per litre (umol/L) | Standard Deviation 0.32 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine | Creatinine | 4.3 micromoles per litre (umol/L) | Standard Deviation 11.02 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
Change from baseline to Day 180 in protein and albumin are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin | Protein | 0.4 grams per litre (g/L) | Standard Deviation 4.47 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin | Albumin | 0.5 grams per litre (g/L) | Standard Deviation 3.22 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin | Protein | 1.5 grams per litre (g/L) | Standard Deviation 6.27 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin | Albumin | 1.2 grams per litre (g/L) | Standard Deviation 3.63 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea
Change from baseline to Day 180 in sodium, chloride, potassium and urea are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data and number analysed signifies participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Sodium | 0.4 millimoles per litre (mmol/L) | Standard Deviation 2.44 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Chloride | 0.6 millimoles per litre (mmol/L) | Standard Deviation 3.3 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Potassium | -0.04 millimoles per litre (mmol/L) | Standard Deviation 0.425 |
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Urea | 0.01 millimoles per litre (mmol/L) | Standard Deviation 0.895 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Urea | 0.40 millimoles per litre (mmol/L) | Standard Deviation 1.197 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Sodium | 0.2 millimoles per litre (mmol/L) | Standard Deviation 3.03 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Potassium | 0.05 millimoles per litre (mmol/L) | Standard Deviation 0.36 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea | Chloride | -0.4 millimoles per litre (mmol/L) | Standard Deviation 4.08 |
Secondary
Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6
Change from baseline to Day 180 in vitamin B6 is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6 | 34.60 nanomoles per litre (nmol/L) | Standard Deviation 99.278 |
| Placebo | Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6 | -215.73 nanomoles per litre (nmol/L) | Standard Deviation 337.775 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes
Change from baseline to Day 180 in basophils/leukocytes is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes | 0.0 Percentage of basophils/leukocytes | Standard Deviation 0.77 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes | 0.1 Percentage of basophils/leukocytes | Standard Deviation 0.74 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte
Change from baseline to Day 180 in eosinophils/leukocytes is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte | 0.4 Percentage of eosinophils/leukocytes | Standard Deviation 1.56 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte | 0.9 Percentage of eosinophils/leukocytes | Standard Deviation 1.66 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes
Change from baseline to Day 180 in erythrocytes is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes | -0.04 10^12 cells per liter | Standard Deviation 0.365 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes | 0.02 10^12 cells per liter | Standard Deviation 0.23 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume
Change from baseline to Day 180 in ery. mean corpuscular volume and mean platelet volume are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume | Ery. mean corpuscular volume | 0.0 femtoliter (fL) | Standard Deviation 2.31 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume | Mean platelet volume | -0.03 femtoliter (fL) | Standard Deviation 0.864 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume | Ery. mean corpuscular volume | 1.1 femtoliter (fL) | Standard Deviation 3.57 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume | Mean platelet volume | 0.61 femtoliter (fL) | Standard Deviation 0.547 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin
Change from baseline to Day 180 in erythrocytes mean corpuscular hemoglobin is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin | -0.1 picogram (pg) | Standard Deviation 1.37 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin | -0.1 picogram (pg) | Standard Deviation 0.99 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit
Change from baseline to Day 180 in hematocrit is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit | -0.004 Litre/litre (L/L) | Standard Deviation 0.0271 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit | 0.006 Litre/litre (L/L) | Standard Deviation 0.0288 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration
Change from baseline to Day 180 in hemoglobin and erythrocytes mean corpuscular hemoglobin concentration are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration | Hemoglobin | -1.7 gram per litre (g/L) | Standard Deviation 8.9 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration | Ery. mean corpuscular hemoglobin concentration | 1.5 gram per litre (g/L) | Standard Deviation 15.1 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration | Hemoglobin | 0.1 gram per litre (g/L) | Standard Deviation 6.62 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration | Ery. mean corpuscular hemoglobin concentration | -4.8 gram per litre (g/L) | Standard Deviation 10.8 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes
Change from baseline to Day 180 in lymphocytes/leukocytes is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes | -1.3 Percentage of lymphocytes/leukocytes | Standard Deviation 10.26 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes | 5.1 Percentage of lymphocytes/leukocytes | Standard Deviation 10.21 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes
Change from baseline to Day 180 in monocytes/leukocytes is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes | 1.0 Percentage of monocytes/leukocytes | Standard Deviation 2.04 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes | 0.9 Percentage of monocytes/leukocytes | Standard Deviation 2.51 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes
Change from baseline to Day 180 in neutrophils/leukocytes is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes | -0.3 Percentage of neutrophils/leukocytes | Standard Deviation 11.45 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes | -7.1 Percentage of neutrophils/leukocytes | Standard Deviation 13.14 |
Secondary
Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils
Change from baseline to Day 180 in reticulocytes, platelets, leukocytes, lymphocytes, monocytes, eosinophils, basophils, neutrophils are presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data and number analysed signifies participants with available data for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Reticulocytes | 0.2 10^12 cells per liter | Standard Deviation 30.08 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Platelets | -11.4 10^12 cells per liter | Standard Deviation 54.5 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Leukocytes | 0.18 10^12 cells per liter | Standard Deviation 1.573 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Lymphocytes | -0.03 10^12 cells per liter | Standard Deviation 0.621 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Monocytes | 0.07 10^12 cells per liter | Standard Deviation 0.085 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Eosinophils | 0.03 10^12 cells per liter | Standard Deviation 0.072 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Basophils | 0.01 10^12 cells per liter | Standard Deviation 0.062 |
| DCR-PHXC | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Neutrophils | 0.14 10^12 cells per liter | Standard Deviation 1.195 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Neutrophils | -1.07 10^12 cells per liter | Standard Deviation 1.7 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Reticulocytes | -23.1 10^12 cells per liter | Standard Deviation 32.21 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Monocytes | -0.02 10^12 cells per liter | Standard Deviation 0.103 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Platelets | 2.3 10^12 cells per liter | Standard Deviation 53.3 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Basophils | -0.04 10^12 cells per liter | Standard Deviation 0.097 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Leukocytes | -1.03 10^12 cells per liter | Standard Deviation 1.931 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Eosinophils | 0.04 10^12 cells per liter | Standard Deviation 0.126 |
| Placebo | Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils | Lymphocytes | 0.06 10^12 cells per liter | Standard Deviation 0.331 |
Secondary
Change From Baseline in Clinical Urinalysis Laboratory Tests: pH
Change from baseline to Day 180 in urine pH is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Urinalysis Laboratory Tests: pH | 0.29 pH | Standard Deviation 1.067 |
| Placebo | Change From Baseline in Clinical Urinalysis Laboratory Tests: pH | -0.10 pH | Standard Deviation 0.738 |
Secondary
Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity
Change from baseline to Day 180 in urine specific gravity is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity | 0.0000 gram per millilitre (g/mL) | Standard Deviation 0.00872 |
| Placebo | Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity | -0.0008 gram per millilitre (g/mL) | Standard Deviation 0.0048 |
Secondary
Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval
Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QTcB interval | -0.8 milliseconds (msec) | Standard Deviation 15.6 |
| DCR-PHXC | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QRS duration | 1.3 milliseconds (msec) | Standard Deviation 6.01 |
| DCR-PHXC | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QTcF interval | -1.1 milliseconds (msec) | Standard Deviation 15.8 |
| DCR-PHXC | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | PR interval | -0.5 milliseconds (msec) | Standard Deviation 13.92 |
| DCR-PHXC | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | RR interval | -7.6 milliseconds (msec) | Standard Deviation 99.35 |
| DCR-PHXC | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QT interval | -2.4 milliseconds (msec) | Standard Deviation 23.73 |
| Placebo | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | RR interval | 17.8 milliseconds (msec) | Standard Deviation 109.73 |
| Placebo | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | PR interval | 1.3 milliseconds (msec) | Standard Deviation 8.14 |
| Placebo | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QRS duration | -1.5 milliseconds (msec) | Standard Deviation 7.53 |
| Placebo | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QTcB interval | -0.7 milliseconds (msec) | Standard Deviation 17.31 |
| Placebo | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QTcF interval | 1.2 milliseconds (msec) | Standard Deviation 15.59 |
| Placebo | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | QT interval | 4.0 milliseconds (msec) | Standard Deviation 22.97 |
Secondary
Change From Baseline in Electrocardiogram (ECG): Heart Rate
Change from baseline in heart rate is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Electrocardiogram (ECG): Heart Rate | 0.4 beats per minute (beats/min) | Standard Deviation 8.36 |
| Placebo | Change From Baseline in Electrocardiogram (ECG): Heart Rate | -1.8 beats per minute (beats/min) | Standard Deviation 9.18 |
Secondary
Change From Baseline in Vital Signs: Body Mass Index (BMI)
Change from baseline to Day 180 in BMI is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Body Mass Index (BMI) | 0.23 Kilograms per square meter (kg/m^2) | Standard Deviation 1.175 |
| Placebo | Change From Baseline in Vital Signs: Body Mass Index (BMI) | 0.14 Kilograms per square meter (kg/m^2) | Standard Deviation 1.293 |
Secondary
Change From Baseline in Vital Signs: Heart Rate
Change from baseline to Day 180 in heart rate is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Heart Rate | 0.8 beats/minute | Standard Deviation 10.64 |
| Placebo | Change From Baseline in Vital Signs: Heart Rate | -3.1 beats/minute | Standard Deviation 6.35 |
Secondary
Change From Baseline in Vital Signs: Height
Change from baseline to Day 180 in height is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Height | 0.70 centimetres (cm) | Standard Deviation 2.383 |
| Placebo | Change From Baseline in Vital Signs: Height | 1.08 centimetres (cm) | Standard Deviation 1.446 |
Secondary
Change From Baseline in Vital Signs: Oral Body Temperature
Change from baseline to Day 180 in oral body temperature is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Oral Body Temperature | 0.01 Degree Celsius (C) | Standard Deviation 0.399 |
| Placebo | Change From Baseline in Vital Signs: Oral Body Temperature | 0.05 Degree Celsius (C) | Standard Deviation 0.446 |
Secondary
Change From Baseline in Vital Signs: Respiratory Rate
Change from baseline to Day 180 in respiratory rate is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Respiratory Rate | -0.2 Breaths per minute | Standard Deviation 3.02 |
| Placebo | Change From Baseline in Vital Signs: Respiratory Rate | -1.1 Breaths per minute | Standard Deviation 5 |
Secondary
Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
Change from baseline to Day 180 in systolic and diastolic blood pressure is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure | Systolic blood pressure | -2.0 millimetres of mercury (mmHg) | Standard Deviation 13.64 |
| DCR-PHXC | Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure | Diastolic blood pressure | 0.8 millimetres of mercury (mmHg) | Standard Deviation 9.2 |
| Placebo | Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure | Systolic blood pressure | -3.0 millimetres of mercury (mmHg) | Standard Deviation 9.82 |
| Placebo | Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure | Diastolic blood pressure | -2.6 millimetres of mercury (mmHg) | Standard Deviation 11.84 |
Secondary
Change From Baseline in Vital Signs: Weight
Change from baseline to Day 180 in weight is presented.
Time frame: Baseline, Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Change From Baseline in Vital Signs: Weight | 1.134 kilograms | Standard Deviation 3.4794 |
| Placebo | Change From Baseline in Vital Signs: Weight | 1.350 kilograms | Standard Deviation 3.433 |
Secondary
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
The Cmax was defined as the maximum observed plasma concentration during a dosing interval. Data for this endpoint is reported only for adults and adolescent participants from PK population.
Time frame: For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours (hrs) postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
Population: The Pharmacokinetic (PK) population included all participants in the safety population without major dosing violations, where safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data and number analysed signifies participants with available data for each specified category.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| DCR-PHXC | Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC | Day 150 | 648 nanogram per millilitre (ng/mL) | Geometric Coefficient of Variation 55.5 |
| DCR-PHXC | Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC | Day 1 | 778 nanogram per millilitre (ng/mL) | Geometric Coefficient of Variation 35.7 |
| DCR-PHXC | Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC | Day 30 | 774 nanogram per millilitre (ng/mL) | Geometric Coefficient of Variation 46.1 |
| Placebo | Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC | Day 1 | 363 nanogram per millilitre (ng/mL) | Geometric Coefficient of Variation 76.7 |
| Placebo | Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC | Day 30 | 350 nanogram per millilitre (ng/mL) | Geometric Coefficient of Variation 48.6 |
Secondary
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Number of participants who had most abnormal post-baseline shift in physical examination are presented. Physical examination shifts were categories into 4 categories: 1) missing; 2) normal; 3) abnormal-not clinically significant (NCS) and 4) abnormal-clinically significant (CS). Each category was presented according body systems including: 1) eyes, ears, nose and throat; 2) chest/respiratory; 3) heart/cardiovascular; 4) gastrointestinal/liver; 5) musculoskeletal/extremities; 6) dermatological/skin; 7) thyroid/neck; 8) lymph nodes; 9) neurological.
Time frame: Baseline up to Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | missing | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | normal | 21 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | abnormal-NCS | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | abnormal-CS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | missing | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | normal | 23 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | abnormal-NCS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | abnormal-CS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | missing | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | normal | 22 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | abnormal-NCS | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | abnormal-CS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | missing | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | normal | 22 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | abnormal-NCS | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | abnormal-CS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | missing | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | normal | 22 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | abnormal-NCS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | abnormal-CS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | missing | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | normal | 18 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | abnormal-NCS | 4 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | abnormal-CS | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | missing | 1 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | normal | 22 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | abnormal-NCS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | abnormal-CS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | missing | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | normal | 23 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | abnormal-NCS | 0 Participants |
| DCR-PHXC | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | missing | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | missing | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | normal | 11 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | missing | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | abnormal-NCS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | normal | 10 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Eyes, ears, nose and throat | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | missing | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | missing | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | abnormal-NCS | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | normal | 12 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | normal | 10 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | abnormal-NCS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Musculoskeletal/extremities | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Chest/respiratory | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | abnormal-NCS | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | missing | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | missing | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | normal | 12 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | abnormal-NCS | 1 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | abnormal-NCS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | normal | 8 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Heart/cardiovascular | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Lymph nodes | normal | 10 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | missing | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | abnormal-NCS | 4 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | normal | 8 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Thyroid/neck | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | abnormal-NCS | 4 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Dermatological/skin | abnormal-CS | 0 Participants |
| Placebo | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Gastrointestinal/liver | abnormal-CS | 0 Participants |
Secondary
Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
Number of TEAEs and TESAEs are presented. An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE was defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalisation, results in persistent disability/incapacity or is a congenital anomaly/birth defect. TEAE was defined as any AE with an onset date/time on or after administration (including any partial administration) of the first dose of study intervention and through the study completion date from the end of study case report form (CRF).
Time frame: From Baseline up to Day 180
Population: The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| DCR-PHXC | Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | TEAEs | 101 Events |
| DCR-PHXC | Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | Serious TEAEs | 1 Events |
| Placebo | Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | TEAEs | 54 Events |
| Placebo | Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | Serious TEAEs | 3 Events |
Secondary
Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits
Percentage of participants whose 24-hour Uox values normalized or near-normalized on at least 2 consecutive visits are presented. Normalization of Uox was defined as less than (\<) 0.46 millimole per 24 hours (mmol/24 hours) and near normalization was defined as greater than or equal to (\>=) 0.46 to \< 0.60 mmol/24 hours (values adjusted per 1.73 square meter \[1.73 m\^2\] body surface area \[BSA\] in participants aged \<18 years).
Time frame: From Day 90 to 180
Population: Modified Intent-to-treat population (MITT) included all participants in the intent-to-treat (ITT) population who had at least one efficacy assessment after the Day 90 dosing visit where ITT population included all participants who were randomized and had at least one post-baseline efficacy assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| DCR-PHXC | Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | 50 Percentage of participants |
| Placebo | Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | 0 Percentage of participants |
Secondary
Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)
Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented.
Time frame: Baseline, Day 180
Population: Analysis population included all adult participants from ITT population who were randomised and had at least one post-baseline efficacy assessment. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| DCR-PHXC | Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | -25.00 Percent change in plasma oxalate |
| Placebo | Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | -0.00 Percent change in plasma oxalate |
Secondary
Percent Change From Baseline to Day 180 in the Number of Kidney Stones
Percent change from baseline to Day 180 in the number of kidney stones is presented.
Time frame: Baseline, Day 180
Population: The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| DCR-PHXC | Percent Change From Baseline to Day 180 in the Number of Kidney Stones | 0.00 Percent change in kidney stone numbers |
| Placebo | Percent Change From Baseline to Day 180 in the Number of Kidney Stones | 0.00 Percent change in kidney stone numbers |
Secondary
Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones
Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm\^2) of kidney stones is presented.
Time frame: Baseline, Day 180
Population: The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment. Number of participants analysed signifies participants with available data.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| DCR-PHXC | Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | -2.13 Percent change in summed surface area |
| Placebo | Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | 21.77 Percent change in summed surface area |
Secondary
Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180
Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation.
Time frame: Baseline, Day 180
Population: The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DCR-PHXC | Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180 | 0.3533 mL/minute/1.73 m^2 | Standard Error 0.3961 |
| Placebo | Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180 | 1.1008 mL/minute/1.73 m^2 | Standard Error 0.54849 |