NCT06860672终止1 期
Safety, Tolerability and Preliminary Efficacy Study of a Single Intrathecal Injection of the Dual Vector AAV-CHD3-R1025W Base Editor for the Treatment of Developmental Disorders Caused by the R1025W Mutation in the CHD3 Gene
Yongguo Yu1 个研究点 分布在 1 个国家目标入组 1 人开始时间: 2025年2月19日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Incidence of drug-related serious adverse events
研究概览
简要总结
To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in the CHD3 gene
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
入排标准
- 年龄范围
- 2 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Snijders Blok-Campeau syndrome
- •Heterozygous mutation of c.3073C>T, p.(Arg1025Trp) in the CHD3 gene
- •Normal liver, heart and immune function
- •Normal coagulation and platelet counts
排除标准
- •Brain tumor or intracranial space-occupying lesion
- •Contraindications to administration of lumbar puncture or sheath injection administration
- •Persistent status epilepticus or recurrent epileptic control instability
- •Presence of unstable systemic disease including active bacterial, fungal or HIV, hepatitis A, hepatitis B infection
- •Serum anti-AAV neutralizing antibody titer >1:50 (ELISA immunoassay)
- •Treatment with immunological agents other than protocol-specified prophylaxis within 3 months
- •Prior gene therapy
- •Participation in another clinical trial, or treatment with another investigational product within 30 days or 5 half-lives
- •Known allergy to any investigational product
研究组 & 干预措施
Dual vector treatment group
Experimental
干预措施: Dual vector DNA base editor (Genetic)
结局指标
主要结局
Incidence of drug-related serious adverse events
时间窗: 0-26 weeks
次要结局
- Evaluate the changes using the Clinical Global impression Scale -Overall improvement (CGI-I)(0-26 weeks)
- Evaluate the changes in the Patient's Global Impressions of Improvement (PGI-I) scale(0-26 weeks)
研究者
Yongguo Yu
Chief physician
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
研究点 (1)
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