| Primary | Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups | This outcome measure reported percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed primary hyperoxaluria type 1 (PH1), primary hyperoxaluria type 2 (PH2), or primary hyperoxaluria type 3 (PH3) subgroups. | The MITT Population included all participants who received at least 1 dose of study intervention and have at least 1 post-baseline spot urinary oxalate to creatinine ratio. Here 'Number of participant analyzed' signified 'Overall number of participants analyzed' and 'Number Analyzed' signifies number of participants with available data for particular timepoint, for the respective arms. | Posted | | Mean | Standard Deviation | Percent change | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
| | | Title | Denominators | Categories |
|---|
| PH1 | - ParticipantsOG0003
- ParticipantsOG0019
- ParticipantsOG0028
| |
| |
| Primary | Absolute Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups | This outcome measure reported absolute change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups. | The MITT Population included all participants who received at least 1 dose of study intervention and have at least 1 post-baseline spot urinary oxalate to creatinine ratio. Here 'Number of participant analyzed' signified 'Overall number of participants analyzed' and 'Number Analyzed' signifies number of participants with available data for particular timepoint, for the respective arms. | Posted | | Mean | Standard Deviation | micromole/millimole | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | This outcome measure reported number of incidents of TEAEs and SAEs. 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 is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and medical events. An AE will be defined as treatment emergent if they have an onset or worsen in severity after a participant receives the study intervention. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Number | | Events | | From baseline (Week 0) up to Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Number of Treatment Emergent Adverse Events and Serious Adverse Events-Nature | This outcome measure reported nature of TEAEs and SAEs. An AE is any untoward medical occurrence in clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and medical events. An AE is treatment emergent if they have an onset or worsen in severity after a participant receives the study intervention. TEAEs are considered as leading to discontinuation if the action taken is marked as "drug withdrawn" on the case report form. TEAEs of special interest include injection site reactions, muscle pain and weakness, and kidney stone events. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Number | | Events | | From baseline (Week 0) up to Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years |
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| Secondary | Change From Baseline in 12-lead Electrocardiogram (ECG)- ECG Mean Heart Rate | This outcome measure reported change from baseline to Month 6 in ECG mean heart rate. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | beats/minute | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in 12-lead ECG- RR Interval | This outcome measure reported change from baseline to Month 6 in RR Interval. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | seconds | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in 12-lead ECG-PR Interval, Aggregate | This outcome measure reported change from baseline to Month 6 in aggregate PR Interval. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | milliseconds | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in 12-lead ECG-QRS Duration, Aggregate | This outcome measure reported change from baseline to Month 6 in aggregate QRS Interval. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | milliseconds | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in 12-lead ECG-QT Interval, Aggregate | This outcome measure reported change from baseline to Month 6 in aggregate QT Interval. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | milliseconds | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in 12-lead ECG-QTcF Interval, Aggregate | This outcome measure reported change from baseline to Month 6 in aggregate QTcF Interval. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | milliseconds | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Participants With Significant Findings- Physical Examination | This outcome measure reported change from baseline to Month 6 in number of participants with significant findings (physical examination). Change from baseline was calculated using formula: value at current time point - baseline value. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Count of Participants | | Participants | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment- Height | This outcome measure reported change from baseline to Month 6 in participants heights. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | centimeters | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment-Weight | This outcome measure reported change from baseline to Month 6 in participants weights. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | kilograms (kg) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment-Body Mass Index (BMI) | This outcome measure reported change from baseline to Month 6 in participants BMI. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | Kilograms per meter square (kg/m^2) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment-Oral Body Temperature | This outcome measure reported change from baseline to Month 6 in participants oral body temperature. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | degree celcius (˚C) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment-Respiratory Rate | This outcome measure reported change from baseline to Month 6 in participants respiratory rate. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | breaths/minute | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment-Heart Rate | This outcome measure reported change from baseline to Month 6 in participants heart rate. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | beats/minute | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Vital Sign Assessment-Systolic Blood Pressure and Diastolic Blood Pressure | This outcome measure reported change from baseline to Month 6 in participants systolic and diastolic blood pressure. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | Millimeters of Mercury (mmHg) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Erythrocytes | This outcome measure reported change from baseline to Month 6 in participants erythrocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Tera cells per liter (10^12 cells /L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Hemoglobin | This outcome measure reported change from baseline to Month 6 in participants hemoglobin. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Grams per deciliter (g/dL) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Hematocrit | This outcome measure reported change from baseline to Month 6 in participants hematocrit. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Ratio of Hematocrit | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Volume | This outcome measure reported change from baseline to Month 6 in participants erythrocytes mean corpuscular volume. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Femtoliter (fL) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin | This outcome measure reported change from baseline to Month 6 in participants erythrocytes mean corpuscular hemoglobin. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | picograms | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | |
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| Secondary | Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin Concentration | This outcome measure reported change from baseline to Month 6 in participant's erythrocytes mean corpuscular hemoglobin concentration. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | g/dL | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | |
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| Secondary | Change From Baseline in Hematology Assessment: Reticulocytes | This outcome measure reported change from baseline to Month 6 in participants reticulocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Percentage of Reticulocytes | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Haematology Assessment: Platelets | This outcome measure reported change from baseline to Month 6 in participants platelets. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Giga cells per liter (10^9 cells /L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Mean Platelet Volume | This outcome measure reported change from baseline to Month 6 in participants mean platelet volume. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | fL | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Leukocytes | This outcome measure reported change from baseline to Month 6 in participants leukocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | 10^9 cells /L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
|
| Secondary | Change From Baseline in Hematology Assessment: Lymphocytes | This outcome measure reported change from baseline to Month 6 in participants lymphocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | 10^9 cells /L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Monocytes | This outcome measure reported change from baseline to Month 6 in participants monocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | 10^9 cells /L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Eosinophils | This outcome measure reported change from baseline to Month 6 in participants eosinophils. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | 10^9 cells /L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Basophils | This outcome measure reported change from baseline to Month 6 in participants basophils. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | 10^9 cells /L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Neutrophils | This outcome measure reported change from baseline to Month 6 in participants neutrophils. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | 10^9 cells /L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Lymphocytes/Leukocytes | This outcome measure reported change from baseline to Month 6 in the ratio of lymphocytes/leukocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Ratio of Lymphocytes/Leukocytes | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Monocytes/Leukocytes | This outcome measure reported change from baseline to Month 6 in the ratio of monocytes/leukocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Ratio of Monocytes/Leukocytes | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Eosinophils/Leukocytes | This outcome measure reported change from baseline to Month 6 in the ratio of eosinophils/leukocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Ratio of Eosinophils/Leukocytes | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
|
| Secondary | Change From Baseline in Hematology Assessment: Basophils/Leukocytes | This outcome measure reported change from baseline to Month 6 in the ratio of basophils/leukocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Ratio of Basophils/Leukocytes | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Hematology Assessment: Neutrophils/Leukocytes | This outcome measure reported change from baseline to Month 6 in the ratio of neutrophils/leukocytes. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Ratio of Neutrophils/Leukocytes | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Alanine Aminotransferase | This outcome measure reported change from baseline to Month 6 in alanine aminotransferase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Units per liter (U/L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Aspartate Aminotransferase | This outcome measure reported change from baseline to Month 6 in aspartate aminotransferase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | U/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Lactate Dehydrogenase | This outcome measure reported change from baseline to Month 6 in lactate dehydrogenase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | U/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Glutamate Dehydrogenase | This outcome measure reported change from baseline to Month 6 in glutamate dehydrogenase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | U/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Gamma Glutamyl Transferase | This outcome measure reported change from baseline to Month 6 in gamma glutamyl transferase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | U/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Alkaline Phosphatase | This outcome measure reported change from baseline to Month 6 in alkaline phosphatase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | U/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Bilirubin and Direct Bilirubin | This outcome measure reported change from baseline to Month 6 in bilirubin and direct bilirubin. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participant analyzed' signified 'Overall number of participants analyzed' and 'Number Analyzed' signifies number of participants with available data for particular timepoint, for the respective arms. | Posted | | Mean | Standard Deviation | micromole per liter (umol/L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Protein | This outcome measure reported change from baseline to Month 6 in protein. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | Grams per liter (g/L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
|
| Secondary | Change From Baseline in Clinical Chemistry Parameter: Albumin | This outcome measure reported change from baseline to Month 6 in albumin. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | g/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Creatine Kinase | This outcome measure reported change from baseline to Month 6 in creatine kinase. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | U/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Sodium | This outcome measure reported change from baseline to Month 6 in sodium. | Safety population included all participants who received at least 1 dose of study intervention. | Posted | | Mean | Standard Deviation | millimole per liter (mmol/L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Chloride | This outcome measure reported change from baseline to Month 6 in chloride. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | mmol/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Potassium | This outcome measure reported change from baseline to Month 6 in potassium. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | mmol/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Creatinine | This outcome measure reported change from baseline to Month 6 in creatinine. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | umol/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Blood Urea Nitrogen | This outcome measure reported change from baseline to Month 6 in blood urea nitrogen. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | mmol/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Cystatin C | This outcome measure reported change from baseline to Month 6 in blood urea Cystatin C. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participants analyzed' signified number of participant with available data for particular timepoint, in the respective arms. | Posted | | Mean | Standard Deviation | milligrams per liter (mg/L) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | All participants that included children (6 to 11 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in Clinical Chemistry Parameter: Plasma Oxalate | This outcome measure reported change from baseline to Month 6 in blood urea plasma oxalate. | Safety population included all participants who received at least 1 dose of study intervention. Here 'Number of participant analyzed' signified 'Overall number of participants analyzed' and 'Number Analyzed' signifies number of participants with available data for particular timepoint, for the respective arms. | Posted | | Mean | Standard Deviation | umol/L | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. | | OG002 | Children 6 to 11 Years | |
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| Secondary | Plasma Pharmacokinetic (PK) Parameter: Maximum Observed Concentration (Cmax), if Estimable | This outcome measure, if estimable, was expected to report Cmax which is defined as maximum observed concentration. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate Cmax, at least one data point prior to Tmax and one data point post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although Cmax was not estimated in this study, the PK data collected in this study will be used in a population PK analysis in the future. | PK population: All participants who received at least one dose of the study intervention and had at least one evaluable post dose PK assessment. Due to only 2 PK concentration - time data (0 to 4 hrs and 4 to 24 hrs) being available within 24 hours post dose in each participant, Cmax was not calculated. Although the protocol allows for calculation of this parameter if, estimable, reliable estimation was not possible in this study. Consequently, Cmax was not reported in the outcomes. | Posted | | Mean | Standard Deviation | nanograms per milliliter (ng/mL) | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. |
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| Secondary | Plasma PK Parameters: Area Under the Concentration-time Curve Calculated to the Last Observable Concentration at Time t (AUCt), if Estimable | This outcome measure, if estimable, was expected to report AUCt which is defined as area under the concentration-time curve calculated from time zero to the last observable concentration at time t. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate AUCt, at least one data point prior to Tmax and three data points post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although AUCt was not estimated in this study, the PK data collected in this study shall be used in a population PK analysis in the future. | PK population: All participants who received at least one dose of study intervention and had at least one evaluable post dose PK assessment. Due to only 2 PK concentration-time data (0 to 4hrs and 4 to 24hrs) being available within 24 hours post dose in each participant, AUCt was not calculated. Although the protocol allows for calculation of AUCt if estimable, reliable estimation was not possible in this study. Consequently, AUCt was not reported in the outcomes. | Posted | | Mean | Standard Deviation | hour*nanograms per milliliter | | Day 1: Postdose 0- to 4-hour and 4- to 24-hour, Days 2, 30, and 90: post dose, Day 150: predose and Day 150: postdose: 0- to 4-hour and 4- to 24-hour | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | |
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| Secondary | Plasma PK Parameters: Area Under the Concentration-time Curve From Time Zero to Infinity (AUC∞), if Estimable | This outcome measure, if estimable, was expected to report AUC∞ which is defined as area under the concentration-time curve calculated to the last observable concentration at time t. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate AUC∞, at least one data point prior to Tmax and three data points post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although AUC∞ was not estimated in this study, the PK data collected in this study will be used in a population PK analysis in the future. | PK population: All participants who received at least one dose of study intervention and had at least one evaluable post dose PK assessment. Due to only 2 PK concentration-time data (0 to 4hrs and 4 to 24hrs) being available within 24 hours post dose in each participant, AUC∞ was not calculated. Although the protocol allows for calculation of AUC∞ if estimable, reliable estimation was not possible in this study. Consequently, AUC∞ was not reported in the outcomes. | Posted | | Mean | Standard Deviation | hour*nanograms per milliliter | | Day 1: Postdose 0- to 4-hour and 4- to 24-hour, Days 2, 30, and 90: post dose, Day 150: predose and Day 150: post-dose: 0- to 4-hour and 4- to 24-hour | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | |
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| Secondary | Percentage of Participants With Spot Urinary Oxalate-to-Creatinine Ratio <=Upper Limit of Normal (ULN) or <=1.5*ULN at Any Time Point Through Month 6 in PH1, PH2, or PH3 Participant Subgroups | This outcome measure reported percentage of participants from baseline to Month 6 Oxalate-to-creatinine Ratio less than and equal to (<=) ULN or <=1.5*ULN at any time point through Month 6 in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups. | The MITT Population included all participants who received at least 1 dose of study intervention and have at least 1 post-baseline spot urinary oxalate to creatinine ratio. Here 'Number of participant analyzed' signifies 'Overall number of participants analyzed' and 'Number analyzed' signifies number of participants with available data for particular timepoint, for the respective arms. | Posted | | Number | | Percentage of participants | | From baseline (week 0) through Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years | All participants that included infants (2 to <6 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 mg/kg, not to exceed 170 mg), subcutaneously from Day 1 through Month 6. |
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| Secondary | Change From Baseline in eGFR at Month 6 (Only in Participants >=12 Months of Age at Screening) in PH1, PH2, or PH3 Participant Subgroups | This outcome measure reported Change from Baseline in GFR estimated Cystatin C at Month 6 (only in participants >=12 Months of age at Screening) in PH1, PH2, or PH3 participant subgroups. The eGFR was calculated using multivariate Schwartz equation using formula: eGFR=39.8*[ht/Scr]^0.456 [1.8/cysC]^0.418 [30/BUN]^0.0791.076^male[ht/1.4]^0.179 where ht (height) = meters, Scr (serum creatinine) = milligrams per deciliter (mg/dL), cysC (cystatin C) = mg/L, and BUN (blood urea nitrogen) = mg/dL. | MITT Population included all participants who received at least 1 dose of study intervention and have at least 1 post-baseline spot urinary oxalate to creatinine ratio. Here 'Number of participant analyzed' signifies 'Overall number of participants analyzed' and 'Number analyzed' signifies number of participants with available data for particular timepoint, for the respective arms. | Posted | | Mean | Standard Deviation | milliliters/minute/1.73m^2 | | Baseline (Week 0), Month 6 | | | | ID | Title | Description |
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| OG000 | Children 0 to <2 Years | All participants that included neonates (0 to less than [<] 2 years) with PH and relatively intact renal function based upon eGFR and serum creatinine, received monthly dose of nedosiran (3.5 milligrams per kilogram [mg/kg], not to exceed 170 milligrams [mg]), subcutaneously from Day 1 through Month 6. | | OG001 | Children 2 to <6 Years |
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