EUCTR2010-019962-10-FRActive, not recruitingNot Applicable
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
Trial Snapshot
- Phase
- Not Applicable
- Status
- Active, not recruiting
- Sponsor
- SANFILIPPO Therapeutics SAS
- Enrollment
- 4
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •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
Exclusion Criteria
- •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®
Investigators
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