Phase 2
N=27
Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
Primary Hyperoxaluria · Primary Hyperoxaluria Type 1 · Primary Hyperoxaluria Type 2 · Primary Hyperoxaluria Type 3
Bottom Line
View on ClinicalTrials.gov: NCT05001269 ↗Enrolled (actual)
27
Serious AEs
18.5%
Results posted
Apr 2026
Primary outcome: Primary: Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups — -74.06; -68.34; -61.44; -17.98 Percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- nedosiran (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Primary completion
- Feb 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups |
-74.06; -68.34; -61.44; -17.98; -16.10; -41.43 | — |
| PRIMARY Absolute Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups |
-563.972; -316.344; -189.106; -64.458; -27.583; -135.283 | — |
| SECONDARY Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
37; 66; 38; 1; 4; 2 | — |
| SECONDARY Number of Treatment Emergent Adverse Events and Serious Adverse Events-Nature |
2; 7; 4; 1; 0; 0 | — |
| SECONDARY Change From Baseline in 12-lead Electrocardiogram (ECG)- ECG Mean Heart Rate |
-11.6; -4.2; 6.2 | — |
| SECONDARY Change From Baseline in 12-lead ECG- RR Interval |
0.1; 0.0; -0.1 | — |
| SECONDARY Change From Baseline in 12-lead ECG-PR Interval, Aggregate |
3.6; 1.8; -1.0 | — |
| SECONDARY Change From Baseline in 12-lead ECG-QRS Duration, Aggregate |
3.2; 0.5; 1.7 | — |
| SECONDARY Change From Baseline in 12-lead ECG-QT Interval, Aggregate |
28.0; 8.2; -6.7 | — |
| SECONDARY Change From Baseline in 12-lead ECG-QTcF Interval, Aggregate |
21.0; 4.5; 1.8 | — |
| SECONDARY Change From Baseline in Participants With Significant Findings- Physical Examination |
0; 0; 1 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment- Height |
6.24; 4.01; 2.76 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment-Weight |
1.130; 1.488; 2.411 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment-Body Mass Index (BMI) |
-0.668; 0.152; 0.608 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment-Oral Body Temperature |
0.36; -0.01; -0.00 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment-Respiratory Rate |
-1.4; -1.8; 0.8 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment-Heart Rate |
6.2; -1.5; 4.7 | — |
| SECONDARY Change From Baseline in Vital Sign Assessment-Systolic Blood Pressure and Diastolic Blood Pressure |
6.8; -6.8; 2.8; 7.6; 1.1; 2.8 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Erythrocytes |
0.195; 0.173; 0.055 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Hemoglobin |
0.575; 0.155; 0.250 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Hematocrit |
0.01; 0.01; 0.00 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Volume |
1.70; -1.25; -0.05 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin |
0.25; -0.48; 0.18 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin Concentration |
-0.42; -0.09; 0.19 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Reticulocytes |
0.15; 0.14; -0.08 | — |
| SECONDARY Change From Baseline in Haematology Assessment: Platelets |
-1.8; -18.6; -27.7 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Mean Platelet Volume |
0.05; -0.10; 0.03 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Leukocytes |
-1.460; 0.383; -0.625 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Lymphocytes |
-1.808; 0.429; -0.577 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Monocytes |
-0.013; -0.024; -0.038 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Eosinophils |
0.078; 0.029; 0.106 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Basophils |
0.035; -0.008; -0.014 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Neutrophils |
-0.165; -0.073; -0.106 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Lymphocytes/Leukocytes |
-10.25; 2.20; -2.51 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Monocytes/Leukocytes |
0.70; -0.45; -0.14 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Eosinophils/Leukocytes |
1.33; 0.22; 1.29 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Basophils/Leukocytes |
0.55; -0.01; -0.09 | — |
| SECONDARY Change From Baseline in Hematology Assessment: Neutrophils/Leukocytes |
7.63; -2.31; 1.50 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Alanine Aminotransferase |
-1.8; 14.9; 5.1 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Aspartate Aminotransferase |
-4.0; 6.4; 7.1 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Lactate Dehydrogenase |
-29.3; 19.5; 1.6 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Glutamate Dehydrogenase |
0.00; 0.28; 0.06 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Gamma Glutamyl Transferase |
-0.4; 1.4; 1.6 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Alkaline Phosphatase |
-6.6; 14.6; -8.4 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Bilirubin and Direct Bilirubin |
-0.46; 1.62; 1.23; -0.07; 0.20; -0.03 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Protein |
-0.8; 0.6; -2.0 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Albumin |
-0.2; 1.2; -0.2 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Creatine Kinase |
-45.6; 9.5; 5.0 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Sodium |
-0.4; -0.6; -0.2 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Chloride |
-1.0; 0.4; -0.9 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Potassium |
-0.42; -0.11; -0.15 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Creatinine |
0.66; 1.10; 1.26 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Blood Urea Nitrogen |
0.56; 0.06; -0.11 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Cystatin C |
-0.120; -0.026; 0.024 | — |
| SECONDARY Change From Baseline in Clinical Chemistry Parameter: Plasma Oxalate |
-9.000; -5.667; -4.400; -2.500; -2.000 | — |
| SECONDARY Plasma Pharmacokinetic (PK) Parameter: Maximum Observed Concentration (Cmax), if Estimable |
NA; NA; NA | — |
| SECONDARY Plasma PK Parameters: Area Under the Concentration-time Curve Calculated to the Last Observable Concentration at Time t (AUCt), if Estimable |
NA; NA; NA | — |
| SECONDARY Plasma PK Parameters: Area Under the Concentration-time Curve From Time Zero to Infinity (AUC∞), if Estimable |
NA; NA; NA | — |
| 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 |
66.7; 44.4; 62.5; 100; 88.9; 100 | — |
| 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 |
0.0; 0.3; -0.8; 3.5; -7.0; 15.0 | — |
Summary
The aim of this study is to evaluate nedosiran in participants 11 years of age and younger who have Primary Hyperoxaluria with relatively intact renal function.
Eligibility Criteria
Inclusion Criteria
- Birth to 11 years of age inclusive, at the time of signing the informed consent.
- Documented diagnosis of PH1 or PH2 or PH3 confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
- Average spot Uox to creatinine ratio at Screening above 2 times the 95th percentile for age (Matos et al, 1999):
- > 0.44 mol/mol in participants 0.34 mol/mol in participants from 6 months to 0.26 mol/mol in participants 12 months to 0.20 mol/mol in participants from 2 to 0.16 mol/mol in participants from 3 to 0.14 mol/mol in participants from 5 to 0.12 mol/mol in participants from 7 to 11 years
- Estimated GFR at Screening ≥ 30 mL/min normalized to 1.73 m2 BSA. See Section 8.2.6.1 for equations. For infants aged less than 12 months, serum creatinine below the 97th percentile of a healthy population (Boer et al., 2010).
- Participants must have been on a stable treatment regimen for PH for 3 months prior to Day 1 and parent(s)/legal guardian should be willing to ensure participant remains on the same stable treatment regimen during the study. Dose adjustments for interval weight gain are acceptable.
- Male or Female
Male participants:
A male participant with a female partner of childbearing potential must agree to use contraception, as detailed in Section 10.5.2, during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.
Female participants:
A female participant is eligible to participate if she is not pregnant (see Section 10.5.1), not breastfeeding, and at least 1 of the following conditions applies:
Not a woman of childbearing potential (WOCBP) as defined in Section 10.5.1 OR A WOCBP who agrees to follow the contraceptive guidance in Section 10.5.2 during the treatment period and for at least 12 weeks after the last dose of study intervention.
Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Note: If the childbearing potential changes after start of the study (e.g., a premenarchal female participant experiences menarche) or the risk of pregnancy changes (e.g., a female participant who is not heterosexually active becomes active), the participant must discuss this with the Investigator, who should determine if a female participant must begin a highly effective method of contraception or a male participant must use a condom. If reproductive status is questionable, additional evaluation should be considered.
- Participant's parent or legal guardian is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
a. For children younger than 12 years of age, assent will be based on local regulation. If assent is required, participant must be able to provide written assent for participation.
- A legal guardian or primary caregiver must be available to help the study-site personnel ensure follow up; accompany the participant to the study site on each assessment day according to the SoA (e.g., able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the rating scales during the scheduled study visits; accurately and reliably dispense study intervention as directed.
- Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)
Exclusion Criteria
- Prior renal or hepatic transplantation; or planned transplantation within the study period
- Currently receiving dialysis or anticipating requirement for dialysis during the study period
- Plasma oxalate (Pox) > 30 μmol/L at Screening
- Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or
Data sourced from ClinicalTrials.gov (NCT05001269). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.