An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.
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
- 4
- 主要终点
- Tolerance and safety
研究概览
简要总结
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the human SGSH and SUMF1 cDNAs for the treatment of Sanfilippo type A syndrome The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.
The primary objective is to assess the tolerance and the safety associated to the proposed treatment through a one-year follow up.
The secondary objective is to collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies.
Four patients will be included in the clinical trial and will be followed during one year.
The enrollment and the follow-up of the patients will take place at Bicêtre Hospital. The Neurosurgery will be performed at Necker-Enfants Malades Hospital.
Safety will be evaluating on clinical, radiological and biological parameters.
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 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: 18 (eighteen) months to end of 6 (six) years
- •Onset of clinical manifestations related to MPSIIIA during the first 5 years of life
- •SGSH activity in peripheral blood cell and / or cultured fibroblast extracts of less than 10% of controls.
- •Patient affiliated to the French social security or assimilated regimens
- •Family understanding the procedure and the informed consent
- •Signed informed consent
- •Vital laboratory parameters within normal range
排除标准
- •Presence of brain atrophy on inclusion MRI judged on a cortico-dural distance of more than 1cm
- •No independent walking (Ability to walk without help)
- •Any condition that would contraindicate permanently anaesthesia
- •Any other permanent medical condition not related to MPSIIIA
- •Any vaccination 1 month before investigational drug administration
- •Intake of aspirin within one month
- •Any medication aiming at modifying the natural course of MPSIIIA given during the 6 months before vector injection
- •Any condition that would contraindicate treatment with Prograf®, Modigraf®, Cellcept® and Solupred®
结局指标
主要结局
Tolerance and safety
时间窗: during the one year follow-up
Measured by * adverse events (by type and severity) * clinical parameters (fever, seizure, headache, abnormal somnolence or lethargy, any new neurological symptoms), * radiological parameters (on MRI, any sign of bleeding after surgery, any hypersignal on T2 weighted images or diffusion images that are not at the points of injection, and any necrotic area evaluated through T1-weighted and diffusion imaging as well as modification of lipids in spectroscopy) * biological parameters (in particular anemia, leucopenia, thrombopenia, liver dysfunction)
次要结局
- To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies(during the one year follow-up)