Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Plain-language summary

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.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

LYSOGENE

Intervention

SAF-301

Start date

5/1/2013

Estimated completion

6/1/2017

Countries

France

Related genes

Locations

• Le Kremlin-Bicêtre, France

Eligibility criteria

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.