A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vectors carrying human NaGlu cDNA for the treatment of Sanfilippo type B syndrome

Institut Pasteur (France)0 个研究点目标入组 4 人开始时间: 2011年8月3日最近更新: 2021年10月11日

适应症

试验速览

2017 Results article in https://pubmed.ncbi.nlm.nih.gov/28713035/ results (added 18/12/2020) 2021 Results article in https://pubmed.ncbi.nlm.nih.gov/34040605/ results (added 30/09/2021)

•1. Age: 18 months to the end of the 5th year
•2. Onset of clinical manifestations related to MPSIIIB during the first 5 years of life
•3. NaGlu activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls
•4. Patient affiliated to a social security regimen
•5. Family understanding the procedure and the informed consent
•6. Vital laboratory parameters within normal range
•Sanfilippo disease is a rare disease and so the countries of recruitment will depend on the availabilities of the patient at the time of recruitment. There will be one study centre in France.

•1. Presence of brain atrophy on pre-inclusion MRI judged on a cortico-dural distance of more than 1 cm
•2. Any condition that would contraindicate permanently anaesthesia
•3. Any other permanent medical condition not related to MPSIIIB
•4. No independent walking (Ability to walk without help)
•5. Any vaccination 1 month before vector injection
•6. Receipt of aspirin within one month
•7. Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection

发起方

Institut Pasteur (France)