EUCTR2017-000506-37-FR进行中（未招募）1 期
CareCN: A phase I/II, open label, escalating dose study to evaluate safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy - CareC
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Genethon
- 入组人数
- 17
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. Patients with severe Crigler-Najjar syndrome requiring phototherapy
- •2. Molecular confirmation of mutation in the UGT1A1 gene by DNA sequencing
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 9
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 7
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •1. Fibrosis score = 3 (METAVIR) or 10 PKa
- •2. Patients who underwent liver transplantation
研究者
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