A Phase 2 Open-Label Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Nedosiran in Pediatric Patients from Birth to 11 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function - PHYOX8

Dicerna Pharmaceuticals Inc0 sites25 target enrollmentStarted: July 12, 2021Last updated: July 22, 2024

Trial Snapshot

No summary available.

•1. Estimated glomerular filtration rate (eGFR) at Screening = 30 mL/min normalized to 1.73 m2 body surface area (BSA).
•2. Average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999:
•> 0.44 mol/mol in participants < 6 months
•> 0.34 mol/mol in participants from 6 months to <12 months
•> 0.26 mol/mol in participants 12 months to < 2 years
•> 0.20 mol/mol in participants from 2 to < 3 years and
•> 0.16 mol/mol in participants from 3 < 5 years
•> 0.14 mol/mol in participants from 5 to < 7 years
•> 0.12 mol/mol in participants from 7 to 11 years
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 25
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Renal or hepatic transplantation (prior or planned within the study period)
•2. Plasma oxalate (Pox) > 30 µmol/L at Screening
•3.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)

Sponsor