Trial Outcomes & Findings for A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (NCT NCT03847909)
NCT ID: NCT03847909
Last Updated: 2024-05-22
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
35 participants
Primary outcome timeframe
From Day 90 to 180
Results posted on
2024-05-22
Participant Flow
The study was conducted at 19 sites in France, Spain, Italy, Netherlands, Germany, United Kingdom, Australia, United States, Canada, Lebanon, and Japan.
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.
Participant milestones
| Measure |
DCR-PHXC
Participants aged greater than or equal to (\>=) 12 years weighing \>=50 kilograms (kg) received nedosiran 170 milligram (mg) (160 milligrams per millilitre \[mg/mL\] free acid equivalent \[FAE\]); participants \>=12 years weighing less than (\<) 50 kg received 136 mg (128 mg FAE); and participants 6-11 years received 3.5 milligrams per kilogram (mg/kg) (3.3 mg/kg FAE) not exceeding 136 mg, subcutaneous (SC) injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
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.
|
|---|---|---|
|
Overall Study
STARTED
|
23
|
12
|
|
Overall Study
Safety Population
|
23
|
12
|
|
Overall Study
Intent to Treat (ITT) Population
|
23
|
12
|
|
Overall Study
Modified ITT (MITT) Population
|
22
|
12
|
|
Overall Study
COMPLETED
|
22
|
11
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
DCR-PHXC
Participants aged greater than or equal to (\>=) 12 years weighing \>=50 kilograms (kg) received nedosiran 170 milligram (mg) (160 milligrams per millilitre \[mg/mL\] free acid equivalent \[FAE\]); participants \>=12 years weighing less than (\<) 50 kg received 136 mg (128 mg FAE); and participants 6-11 years received 3.5 milligrams per kilogram (mg/kg) (3.3 mg/kg FAE) not exceeding 136 mg, subcutaneous (SC) injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
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.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
Baseline Characteristics
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
Baseline characteristics by cohort
| Measure |
DCR-PHXC
n=23 Participants
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.
|
Placebo
n=12 Participants
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.
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
23.7 years
STANDARD_DEVIATION 11.95 • n=99 Participants
|
23.6 years
STANDARD_DEVIATION 11.48 • n=107 Participants
|
23.7 years
STANDARD_DEVIATION 11.62 • n=206 Participants
|
|
Age, Customized
Between 2 to 11 years
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Age, Customized
Between 12 to 17 years
|
6 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
|
Age, Customized
Between 18 to 64 years
|
14 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
30 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
6 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: From Day 90 to 180Population: 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.
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.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
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
|
-1664.4 Percent change in 24-hour Uox AUC
Standard Error 1189.96
|
SECONDARY outcome
Timeframe: From Day 90 to 180Population: 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.
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).
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits
|
50 Percentage of participants
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm\^2) of kidney stones is presented.
Outcome measures
| Measure |
DCR-PHXC
n=16 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones
|
-2.13 Percent change in summed surface area
Interval -100.0 to 228.6
|
21.77 Percent change in summed surface area
Interval -24.3 to 1500.0
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Percent change from baseline to Day 180 in the number of kidney stones is presented.
Outcome measures
| Measure |
DCR-PHXC
n=16 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Percent Change From Baseline to Day 180 in the Number of Kidney Stones
|
0.00 Percent change in kidney stone numbers
Interval -100.0 to 200.0
|
0.00 Percent change in kidney stone numbers
Interval -94.1 to 100.0
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented.
Outcome measures
| Measure |
DCR-PHXC
n=14 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)
|
-25.00 Percent change in plasma oxalate
Interval -61.9 to 22.2
|
-0.00 Percent change in plasma oxalate
Interval -25.0 to 350.0
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment.
Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation.
Outcome measures
| Measure |
DCR-PHXC
n=23 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180
|
0.3533 mL/minute/1.73 m^2
Standard Error 0.39610
|
1.1008 mL/minute/1.73 m^2
Standard Error 0.54849
|
SECONDARY outcome
Timeframe: From Baseline up to Day 180Population: 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 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).
Outcome measures
| Measure |
DCR-PHXC
n=23 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
TEAEs
|
101 Events
|
54 Events
|
|
Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
Serious TEAEs
|
1 Events
|
3 Events
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline in heart rate is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Electrocardiogram (ECG): Heart Rate
|
0.4 beats per minute (beats/min)
Standard Deviation 8.36
|
-1.8 beats per minute (beats/min)
Standard Deviation 9.18
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
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 15.80
|
1.2 milliseconds (msec)
Standard Deviation 15.59
|
|
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
|
1.3 milliseconds (msec)
Standard Deviation 8.14
|
|
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
|
-1.5 milliseconds (msec)
Standard Deviation 7.53
|
|
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
|
4.0 milliseconds (msec)
Standard Deviation 22.97
|
|
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 15.60
|
-0.7 milliseconds (msec)
Standard Deviation 17.31
|
|
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
|
17.8 milliseconds (msec)
Standard Deviation 109.73
|
SECONDARY outcome
Timeframe: Baseline up to Day 180Population: 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 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.
Outcome measures
| Measure |
DCR-PHXC
n=23 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Eyes, ears, nose and throat · missing
|
1 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Eyes, ears, nose and throat · normal
|
21 Participants
|
11 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Eyes, ears, nose and throat · abnormal-NCS
|
1 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Eyes, ears, nose and throat · abnormal-CS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Chest/respiratory · missing
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Chest/respiratory · normal
|
23 Participants
|
12 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Chest/respiratory · abnormal-NCS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Chest/respiratory · abnormal-CS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Heart/cardiovascular · missing
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Heart/cardiovascular · normal
|
22 Participants
|
12 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Heart/cardiovascular · abnormal-NCS
|
1 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Heart/cardiovascular · abnormal-CS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Gastrointestinal/liver · missing
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Gastrointestinal/liver · normal
|
22 Participants
|
8 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Gastrointestinal/liver · abnormal-NCS
|
1 Participants
|
4 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Gastrointestinal/liver · abnormal-CS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Musculoskeletal/extremities · missing
|
1 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Musculoskeletal/extremities · normal
|
22 Participants
|
10 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Musculoskeletal/extremities · abnormal-NCS
|
0 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Musculoskeletal/extremities · abnormal-CS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Dermatological/skin · missing
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Dermatological/skin · normal
|
18 Participants
|
8 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Dermatological/skin · abnormal-NCS
|
4 Participants
|
4 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Dermatological/skin · abnormal-CS
|
1 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Thyroid/neck · missing
|
1 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Thyroid/neck · normal
|
22 Participants
|
10 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Thyroid/neck · abnormal-NCS
|
0 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Thyroid/neck · abnormal-CS
|
0 Participants
|
0 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Lymph nodes · missing
|
0 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Lymph nodes · normal
|
23 Participants
|
10 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Lymph nodes · abnormal-NCS
|
0 Participants
|
1 Participants
|
|
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Lymph nodes · abnormal-CS
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in height is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Height
|
0.70 centimetres (cm)
Standard Deviation 2.383
|
1.08 centimetres (cm)
Standard Deviation 1.446
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in weight is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Weight
|
1.134 kilograms
Standard Deviation 3.4794
|
1.350 kilograms
Standard Deviation 3.4330
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in BMI is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Body Mass Index (BMI)
|
0.23 Kilograms per square meter (kg/m^2)
Standard Deviation 1.175
|
0.14 Kilograms per square meter (kg/m^2)
Standard Deviation 1.293
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in oral body temperature is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Oral Body Temperature
|
0.01 Degree Celsius (C)
Standard Deviation 0.399
|
0.05 Degree Celsius (C)
Standard Deviation 0.446
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in heart rate is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Heart Rate
|
0.8 beats/minute
Standard Deviation 10.64
|
-3.1 beats/minute
Standard Deviation 6.35
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in respiratory rate is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Respiratory Rate
|
-0.2 Breaths per minute
Standard Deviation 3.02
|
-1.1 Breaths per minute
Standard Deviation 5.00
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in systolic and diastolic blood pressure is presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
Systolic blood pressure
|
-2.0 millimetres of mercury (mmHg)
Standard Deviation 13.64
|
-3.0 millimetres of mercury (mmHg)
Standard Deviation 9.82
|
|
Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
Diastolic blood pressure
|
0.8 millimetres of mercury (mmHg)
Standard Deviation 9.20
|
-2.6 millimetres of mercury (mmHg)
Standard Deviation 11.84
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
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.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
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
|
-1.1 Units per litre (U/L)
Standard Deviation 4.59
|
|
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
|
-1.3 Units per litre (U/L)
Standard Deviation 4.73
|
|
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
|
-0.38 Units per litre (U/L)
Standard Deviation 1.064
|
|
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
|
-0.2 Units per litre (U/L)
Standard Deviation 3.03
|
|
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
|
-16.8 Units per litre (U/L)
Standard Deviation 41.78
|
|
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
|
1.0 Units per litre (U/L)
Standard Deviation 20.72
|
|
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
|
-34.1 Units per litre (U/L)
Standard Deviation 160.46
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in bilirubin, direct bilirubin and creatinine are presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
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
|
0.4 micromoles per litre (umol/L)
Standard Deviation 1.80
|
|
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
|
0.1 micromoles per litre (umol/L)
Standard Deviation 0.32
|
|
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
|
4.3 micromoles per litre (umol/L)
Standard Deviation 11.02
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in protein and albumin are presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
Protein
|
0.4 grams per litre (g/L)
Standard Deviation 4.47
|
1.5 grams per litre (g/L)
Standard Deviation 6.27
|
|
Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
Albumin
|
0.5 grams per litre (g/L)
Standard Deviation 3.22
|
1.2 grams per litre (g/L)
Standard Deviation 3.63
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in sodium, chloride, potassium and urea are presented.
Outcome measures
| Measure |
DCR-PHXC
n=22 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=11 Participants
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.
|
|---|---|---|
|
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
|
0.2 millimoles per litre (mmol/L)
Standard Deviation 3.03
|
|
Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea
Chloride
|
0.6 millimoles per litre (mmol/L)
Standard Deviation 3.30
|
-0.4 millimoles per litre (mmol/L)
Standard Deviation 4.08
|
|
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
|
0.05 millimoles per litre (mmol/L)
Standard Deviation 0.360
|
|
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
|
0.40 millimoles per litre (mmol/L)
Standard Deviation 1.197
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in vitamin B6 is presented.
Outcome measures
| Measure |
DCR-PHXC
n=2 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=3 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6
|
34.60 nanomoles per litre (nmol/L)
Standard Deviation 99.278
|
-215.73 nanomoles per litre (nmol/L)
Standard Deviation 337.775
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in erythrocytes is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes
|
-0.04 10^12 cells per liter
Standard Deviation 0.365
|
0.02 10^12 cells per liter
Standard Deviation 0.230
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in hemoglobin and erythrocytes mean corpuscular hemoglobin concentration are presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
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 8.90
|
0.1 gram per litre (g/L)
Standard Deviation 6.62
|
|
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 15.10
|
-4.8 gram per litre (g/L)
Standard Deviation 10.80
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in hematocrit is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit
|
-0.004 Litre/litre (L/L)
Standard Deviation 0.0271
|
0.006 Litre/litre (L/L)
Standard Deviation 0.0288
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in ery. mean corpuscular volume and mean platelet volume are presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
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
|
1.1 femtoliter (fL)
Standard Deviation 3.57
|
|
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
|
0.61 femtoliter (fL)
Standard Deviation 0.547
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in erythrocytes mean corpuscular hemoglobin is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin
|
-0.1 picogram (pg)
Standard Deviation 1.37
|
-0.1 picogram (pg)
Standard Deviation 0.99
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in reticulocytes, platelets, leukocytes, lymphocytes, monocytes, eosinophils, basophils, neutrophils are presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
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
|
-23.1 10^12 cells per liter
Standard Deviation 32.21
|
|
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 54.50
|
2.3 10^12 cells per liter
Standard Deviation 53.30
|
|
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
|
-1.03 10^12 cells per liter
Standard Deviation 1.931
|
|
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
|
0.06 10^12 cells per liter
Standard Deviation 0.331
|
|
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
|
-0.02 10^12 cells per liter
Standard Deviation 0.103
|
|
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
|
0.04 10^12 cells per liter
Standard Deviation 0.126
|
|
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
|
-0.04 10^12 cells per liter
Standard Deviation 0.097
|
|
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
|
-1.07 10^12 cells per liter
Standard Deviation 1.700
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in lymphocytes/leukocytes is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes
|
-1.3 Percentage of lymphocytes/leukocytes
Standard Deviation 10.26
|
5.1 Percentage of lymphocytes/leukocytes
Standard Deviation 10.21
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in monocytes/leukocytes is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes
|
1.0 Percentage of monocytes/leukocytes
Standard Deviation 2.04
|
0.9 Percentage of monocytes/leukocytes
Standard Deviation 2.51
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in eosinophils/leukocytes is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte
|
0.4 Percentage of eosinophils/leukocytes
Standard Deviation 1.56
|
0.9 Percentage of eosinophils/leukocytes
Standard Deviation 1.66
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in basophils/leukocytes is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes
|
0.0 Percentage of basophils/leukocytes
Standard Deviation 0.77
|
0.1 Percentage of basophils/leukocytes
Standard Deviation 0.74
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in neutrophils/leukocytes is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes
|
-0.3 Percentage of neutrophils/leukocytes
Standard Deviation 11.45
|
-7.1 Percentage of neutrophils/leukocytes
Standard Deviation 13.14
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in urine specific gravity is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity
|
0.0000 gram per millilitre (g/mL)
Standard Deviation 0.00872
|
-0.0008 gram per millilitre (g/mL)
Standard Deviation 0.00480
|
SECONDARY outcome
Timeframe: Baseline, Day 180Population: 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.
Change from baseline to Day 180 in urine pH is presented.
Outcome measures
| Measure |
DCR-PHXC
n=21 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=10 Participants
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.
|
|---|---|---|
|
Change From Baseline in Clinical Urinalysis Laboratory Tests: pH
|
0.29 pH
Standard Deviation 1.067
|
-0.10 pH
Standard Deviation 0.738
|
SECONDARY outcome
Timeframe: 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 postdosePopulation: 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.
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.
Outcome measures
| Measure |
DCR-PHXC
n=12 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
Day 1
|
778 nanogram per millilitre (ng/mL)
Geometric Coefficient of Variation 35.7
|
363 nanogram per millilitre (ng/mL)
Geometric Coefficient of Variation 76.7
|
|
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
Day 30
|
774 nanogram per millilitre (ng/mL)
Geometric Coefficient of Variation 46.1
|
350 nanogram per millilitre (ng/mL)
Geometric Coefficient of Variation 48.6
|
|
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
Day 150
|
648 nanogram per millilitre (ng/mL)
Geometric Coefficient of Variation 55.5
|
—
|
SECONDARY outcome
Timeframe: 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 postdosePopulation: 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.
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.
Outcome measures
| Measure |
DCR-PHXC
n=12 Participants
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 free acid equivalent \[FAE\]) not exceeding 136 mg, SC injection into the abdomen or thigh on Days 1, 30, 60, 90, 120 and 150.
|
Placebo
n=5 Participants
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.
|
|---|---|---|
|
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
|
6450 hours*nanograms per millilitre (h*ng/mL)
Geometric Coefficient of Variation 54.5
|
|
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
|
6400 hours*nanograms per millilitre (h*ng/mL)
Geometric Coefficient of Variation 45.1
|
|
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
|
—
|
Adverse Events
DCR-PHXC
Serious events: 1 serious events
Other events: 16 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
DCR-PHXC
n=23 participants at risk
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.
|
Placebo
n=12 participants at risk
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.
|
|---|---|---|
|
Cardiac disorders
Tachycardia
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Blood and lymphatic system disorders
Blood creatinine increased
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Renal and urinary disorders
Renal colic
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
Other adverse events
| Measure |
DCR-PHXC
n=23 participants at risk
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.
|
Placebo
n=12 participants at risk
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.
|
|---|---|---|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
General disorders
Injection site erythema
|
21.7%
5/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
General disorders
Injection site pain
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
General disorders
Fatigue
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
16.7%
2/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Reproductive system and breast disorders
Menstrual disorder
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Psychiatric disorders
Depression
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Investigations
Neutrophil count decreased
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Investigations
Blood creatine phosphokinase increased
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Investigations
Glutamate dehydrogenase increased
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Injury, poisoning and procedural complications
Adverse event following immunisation
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Injury, poisoning and procedural complications
Meniscus injury
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Nervous system disorders
Headache
|
17.4%
4/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
25.0%
3/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Nervous system disorders
Sensory disturbance
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Nausea
|
17.4%
4/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Abdominal pain
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
0.00%
0/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
16.7%
2/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Chronic gastritis
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Renal and urinary disorders
Nephrolithiasis
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
16.7%
2/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Renal and urinary disorders
Renal colic
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Renal and urinary disorders
Hydronephrosis
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.7%
2/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
16.7%
2/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Infections and infestations
Upper respiratory tract infection
|
4.3%
1/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Infections and infestations
COVID-19
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/23 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
8.3%
1/12 • From Day 1 up to end of the study (Day 180)
The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. All serious and non-serious adverse events presented here are treatment emergent adverse events.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee SPONSOR agrees that the Principal Investigator shall have the right to publish or permit the publication of any information or material relating to or arising out of the work after prior submittal to SPONSOR provided that if SPONSOR shall so request, the investigator will delay publication for a maximum of ninety (90) days after submittal to SPONSOR to enable SPONSOR to protect its rights. SPONSOR will comment on such documents within thirty (30) days.
- Publication restrictions are in place
Restriction type: OTHER