NCT05514249Unknown1 期
Treatment of a Single Patient With CRD-TMH-001
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- To assess the safety of CRD-TMH-001
研究概览
简要总结
The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.
详细描述
The objective of the study is to assess the safety and preliminary efficacy of CRD-TMH-001 after intravenous administration for a period of 1 year with long-term follow-up out to 15 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
盲法说明
Single patient clinical trial
入排标准
- 年龄范围
- 18 Years 至 28 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completion of informed consent
- •Confirmation of genetic mutation
- •Confirmation of absence of elevated AAV9 NAbs
排除标准
- •Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.
研究组 & 干预措施
Single patient
Experimental
Single dose of CRD-TMH-001 administered by IV
干预措施: CRD-TMH-001 (Drug)
结局指标
主要结局
To assess the safety of CRD-TMH-001
时间窗: 1 year
To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.
次要结局
未报告次要终点
研究者
研究点 (1)
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