P2-SAF-301 is an open-label interventional study with no administration of investigational product, following the long-term safety and tolerability of intracerebral SAF-301 previously given to 4 patients with Sanfilippo type A syndrome. The primary objective is to collect additional safety and tolerability data; the secondary objective is to assess effects on neurological and psychological status and potential biological markers. Only patients who completed the earlier Phase 1/2 study (P1-SAF-301), with parental informed consent, were enrolled.
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: * Patients having completed the previous phase I/II study (P1-SAF-301), * Family understanding the follow-up procedures and the informed consent, * Patient's parents having signed the informed consent form. Exclusion Criteria: * Participation in any other clinical trial.