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

Summary

P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome. The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome. The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.

Timeline

Start

2013-05

Primary completion

2017-05

Completion

2017-06

Drugs

Indications