NCT02053064已完成1 期
Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- LYSOGENE
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Checking of adverse events
研究概览
简要总结
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.
The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.
The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients having completed the previous phase I/II study (P1-SAF-301),
- •Family understanding the follow-up procedures and the informed consent,
- •Patient's parents having signed the informed consent form.
排除标准
- •Participation in any other clinical trial.
结局指标
主要结局
Checking of adverse events
时间窗: until 60 months after SAF-301 administration
次要结局
- Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments(5 years after the injection of SAF-301)
- Information on changes in potential biomarkers of the disease(5 years after the injection of SAF-301)
- Information to further evaluation of immune response(5 years after the injection of SAF-301)
研究者
研究点 (2)
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