edosiran in Pediatric Patients from Birth to 5 Years of Age with PH1, PH2, or PH3 and Relatively Intact Renal Functio

Phase 1

• Conditions: Primary Hyperoxaluria; MedDRA version: 20.1Level: PTClassification code 10020703Term: HyperoxaluriaSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2021-001083-16-ES
• Lead Sponsor: Dicerna Pharmaceuticals Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-23
• Last Update Posted: 24 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 15

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: 15
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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified