Trial · NCT05001269
Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
NCT05001269, Phase 2, Completed, Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function. Trials, record NCT05001269. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05001269
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
- NCT number
- NCT05001269
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 27 participants, actual
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Conditions
- Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
- Interventions
- nedosiran
- First posted
- 2021-08-11
- Last updated
- 2026-04-16
Source ClinicalTrials.gov
Registered outcomes60
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups | Baseline (Week 0), Month 6 | 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. |
| Primary | Absolute Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups | Baseline (Week 0), Month 6 | 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. |
| Secondary | Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From baseline (Week 0) up to Month 6 | 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. |
| Secondary | Number of Treatment Emergent Adverse Events and Serious Adverse Events-Nature | From baseline (Week 0) up to Month 6 | 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. |
| Secondary | Change From Baseline in 12-lead Electrocardiogram (ECG)- ECG Mean Heart Rate | Baseline (Week 0), Month 6 | This outcome measure reported change from baseline to Month 6 in ECG mean heart rate. |
| Secondary | Change From Baseline in 12-lead ECG- RR Interval | Baseline (Week 0), Month 6 | This outcome measure reported change from baseline to Month 6 in RR Interval. |
| Secondary | Change From Baseline in 12-lead ECG-PR Interval, Aggregate | Baseline (Week 0), Month 6 | This outcome measure reported change from baseline to Month 6 in aggregate PR Interval. |
| Secondary | Change From Baseline in 12-lead ECG-QRS Duration, Aggregate | Baseline (Week 0), Month 6 | This outcome measure reported change from baseline to Month 6 in aggregate QRS Interval. |
| Secondary | Change From Baseline in 12-lead ECG-QT Interval, Aggregate | Baseline (Week 0), Month 6 | This outcome measure reported change from baseline to Month 6 in aggregate QT Interval. |
| Secondary | Change From Baseline in 12-lead ECG-QTcF Interval, Aggregate | Baseline (Week 0), Month 6 | This outcome measure reported change from baseline to Month 6 in aggregate QTcF Interval. |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05001269
GET https://conductscience.org/api/trials/v1/NCT05001269/registered?page=2