NCT05001269

Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH 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 11Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Industry
• Sex: All
• Age: 11 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is an open-label, repeat-dose, Phase 2 study of nedosiran in participants 11 years of age or younger who have PH1, PH2 or PH 3 and relatively intact renal function. Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180. The total duration of this study is approximately 15 months from first participant, first visit, until last participant, last visit.

Conditions

• Primary Hyperoxaluria
• Primary Hyperoxaluria Type 1
• Primary Hyperoxaluria Type 2
• Primary Hyperoxaluria Type 3

Interventions

Type	Name	Description
DRUG	nedosiran	Monthly subcutaneous dosing throughout study period

Timeline

• Start date: 2022-02-22
• Primary completion: 2025-02-05
• Completion: 2025-02-05
• First posted: 2021-08-11
• Last updated: 2026-04-16
• Results posted: 2026-04-16

Locations

13 sites across 11 countries: United States, Canada, Germany, Italy, Japan, Lebanon, Poland, Spain, Turkey (Türkiye), United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study