NL-OMON52580招募中不适用
CareCN: A phase I/II, open-label, study to evaluate the 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 - Care C
Généthon0 个研究点目标入组 5 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •Main Inclusion criteria
- •[1] Patients with severe Crigler-Najjar syndrome requiring daily phototherapy
- •(>= 6h/day)
- •[2] Molecular confirmation of mutations in the UGT1A1 gene by DNA sequencing
排除标准
- •Main Exclusion criteria
- •[1] Fibrosis score >= 3 (METAVIR) or 10 PKa
- •[2] Patients who underwent liver transplantation
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