Phase 1/2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0
研究概览
简要总结
The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms.
Participants will:
Receive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study
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
- 4 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CLN6
- •At least 4 months old
排除标准
- •Presence of another inherited neurologic disease
- •Prior stem cell transplantation
- •Prior gene transfer, gene editing, or viral vector therapy
研究组 & 干预措施
Treatment group
干预措施: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0
时间窗: From informed consent through Month 24
Treatment-emergent adverse events, including serious adverse events, hospitalizations, deaths, clinically significant laboratory abnormalities, and other clinically significant safety findings will be recorded from informed consent through Month 24. Adverse events will be assessed for intensity using CTCAE version 5.0 and for relationship to scAAV9.CB.CLN6. The number and percentage of participants with treatment-emergent adverse events will be summarized.
次要结局
- Change From Baseline in Hamburg Rating Scale Score(Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24)
- Change From Baseline in Weill-Cornell Late Infantile Neuronal Ceroid Lipofuscinosis Scale Score(Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24)