Protocol AMT110-CD-001: A Phase I/II, Open-label, Study of Intracerebral Administration of Adeno-associated Viral Vector Containing the Human Alpha-N-acetylglucosaminidase cDNA in Children With Sanfilippo Type B Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-Related (Serious) Adverse Events as assessed by continuous evaluation of change from baseline
研究概览
简要总结
This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.
详细描述
This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.
Four patients, 18 months up to the 5th birthday, have been included.
The inclusion period will be 8 to 12 months. The duration of follow-up for each patient is 1 year post-surgery. The duration of the first extension phase is 18 months. The duration of the second extension phase is 36 months. Therefore, the maximum time of the follow-up will be 66 months
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
- 18 Months 至 60 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Presence of brain atrophy on baseline MRI judged on a cortico-dural distance of more than 0.6 cm;
- •Any condition that would contraindicate general anesthesia;
- •Any other permanent medical condition not related to MPSIIIB that could contraindicate the study participation;
- •No independent walking (ability to walk without help);
- •Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection (sleep and mood regulators are accepted);
- •Any condition that would contraindicate treatment with Modigraf®, Cellcept® and prednisolone (Solupred® and Solumedrol®).
研究组 & 干预措施
rAAV2/5-hNAGLU
Each patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total).
干预措施: rAAV2/5-hNAGLU (Drug)
结局指标
主要结局
Number of Participants With Treatment-Related (Serious) Adverse Events as assessed by continuous evaluation of change from baseline
时间窗: Baseline until end of study (Month 66)
Multiple measurements will be aggregated to derive the number of participants with Abnormal Laboratory Values and/or Adverse Events that are related to Treatment.
次要结局
- Number of Participants with presence of brain atrophy, white matter lesions and other lesions as assessed by cerebral MRI(Baseline until end of study (Month 66))