This study examined a gene therapy called SAF-301, delivered into the brain, in children with Sanfilippo type A syndrome (MPS IIIA). It enrolled children aged 18 months to 6 years who had early-onset symptoms and low enzyme activity. It was carried out in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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 French social security or assimilated regimens * Family understanding the procedure and the informed consent * Signed informed consent * Vital laboratory parameters within normal range Exclusion Criteria: * 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®