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

Dicerna Pharmaceuticals Inc.0 个研究点目标入组 15 人开始时间: 2021年10月18日最近更新: 2022年3月2日

试验速览

暂无简介。

•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

•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)

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