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临床试验/EUCTR2021-001083-16-IT
EUCTR2021-001083-16-IT进行中(未招募)1 期

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:

试验速览

阶段
1 期
状态
进行中(未招募)
入组人数
15

研究概览

简要总结

暂无简介。

研究设计

研究类型
Interventional clinical trial of medicinal product

入排标准

性别
All

入选标准

  • 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

排除标准

  • 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)

研究者

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