A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy (SMA)
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- long-term safety：Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)
研究概览
简要总结
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
详细描述
This study plans to enroll type 1 SMA patients who have previously received EXG001-307 treatment in the parent trial EXG001-307-102 to observe the long-term safety and efficacy of gene therapy with EXG001-302.
The first year of long-term follow-up will be conducted every 3 months ; In the second year, follow-up visits will be conducted every six months, with telephone follow ups at 15 and 21 months during this period; From 3 to 5 years after gene therapy, follow up by phone every six months and follow on on site once a year.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 1 Month 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Previously received treatment with EXG001-307 in trial EXG001-307-102;
- •Parents or guardians understand the research procedure and sign the ICF; Good compliance and willingness to follow research procedures. Voluntarily participate in this clinical trial.
排除标准
- •Parents or guardians are unwilling or unable to participate in long-term follow-up studies.
结局指标
主要结局
long-term safety：Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)
时间窗: 4 years
Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)
次要结局
- Evaluate the long-term effectiveness of EXG001-307 treatment: assess the achievement of exercise milestones based on the scale(4 years)
- Evaluate the long-term effectiveness of EXG001-307 treatment: evaluate event free survival rate(4 years)
- Evaluate the immunogenicity of EXG001-307 after treatment, including measuring the titers of anti-AAV9-ADA and anti-SMN-ADA antibodies.(4 years)
- Evaluate the distribution of viral vectors after treatment with EXG001-307: detect the level of vector genome in saliva, feces, and urine samples of subjects.(4 years)
- Exploratory objective: To calculate the proportion of subjects receiving other SMA treatments during the trial period(4 years)