AN OPEN-LABEL, SINGLE ARM, MONOCENTRIC, PHASE I/II CLINICAL STUDY OF INTRACEREBRAL ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS SEROTYPE 10 CARRYING THE HUMAN SGSH AND SUMF1 cDNAS FOR THE TREATMENT OF SANFILIPPO TYPE A SYNDROME - Intracerebral Gene therapy for Sanfilippo type A syndrome
- Conditions
- Sanfilippo type A syndrome (also named Mucopolysaccharidosis Type A)MedDRA version: 12.1Level: LLTClassification code 10056918Term: Sanfilippo's syndrome
- Registration Number
- EUCTR2010-019962-10-FR
- Lead Sponsor
- SANFILIPPO Therapeutics SAS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 4
•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 social security
•Family understanding the procedure and the informed consent
•Signed informed consent
•Vital laboratory parameters within normal range
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
•Presence of brain atrophy on pre-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 treatment
•Receipt 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®, Cellcept® and Solupred®
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
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