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A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function

A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function

Status
Active, not recruiting
Phases
Phase 2
Study type
Interventional
Source
LBCTR
Registry ID
LBCTR2021104866
Enrollment
5
Registered
2022-01-27
Start date
2021-12-06
Completion date
Unknown
Last updated
2026-02-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Hyperoxaluria Type 1, 2 & 3. hyperoxaluria

Interventions

monthly SC doses (3.5 mg/kg) of nedosiran over 6 months.
DCR-PHXC 170 mg/mL Solution for Injection
Nedosiran

Sponsors

Dicerna Pharmaceuticals
Lead Sponsor
Premier Research
Collaborator

Eligibility

Sex/Gender
All
Age
No minimum to 5 Years

Inclusion criteria

Inclusion criteria: Key inclusion criteria include • Estimated glomerular filtration rate (eGFR) at Screening = 30 mL/min normalized to 1.73 m2 body surface area (BSA). • Average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999: o > 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 5 years

Exclusion criteria

Exclusion criteria: Key exclusion criteria include • Renal or hepatic transplantation (prior or planned within 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 skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)

Design outcomes

Primary

MeasureTime frame
Name: To characterize the safety of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Timepoints: 6 months;Measure: Change from Baseline in 12-lead ECG, physical examination findings, vital sign assessments, and clinical laboratory tests—

Secondary

MeasureTime frame
Name: To assess the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Timepoints: 6 months;Measure: Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio—

Countries

France, Lebanon, Poland, Turkey, United Kingdom, United States of America

Contacts

Public ContactSarah Kharsa

Clinart MEA

sarah.kharsa@clinart.net+96181209199

Outcome results

None listed

Source: LBCTR (via WHO ICTRP) · Data processed: Feb 7, 2026