EUCTR2021-001083-16-ITActive, not recruitingPhase 1
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 - PHYOX8
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Dicerna Pharmaceuticals Inc.
- Enrollment
- 15
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •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 to 5 years
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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
Exclusion Criteria
- •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)
Investigators
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