EUCTR2012-000856-33-FR
Active, not recruiting
Phase 1
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
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Mucopolysaccharidosis III B
- Sponsor
- Institut Pasteur
- Status
- Active, not recruiting
- Last Updated
- 8 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Age: 18 months up to the 5th birthday;
- •Onset of clinical manifestations related to MPSIIIB;
- •NaGlu activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls;
- •Patient affiliated to, or covered by a French social security regimen or European patients with European Health Insurance Card
- •Family understanding the procedure and the informed consent;
- •Signed informed consent by both parents or legal representative
- •Vital laboratory parameters within normal range
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 4
- •F.1\.2 Adults (18\-64 years) no
Exclusion Criteria
- •Presence of brain atrophy on pre\-inclusion MRI judged on a cortico\-dural distance of more than 0\.6 cm
- •Any condition that would contraindicate general anaesthesia;
- •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 Tacrolimus, Cellcept® and Solupred®/Solumedrol®.
Outcomes
Primary Outcomes
Not specified
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