NCT02053064

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

Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: LYSOGENE · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Mucopolysaccharidosis Type III A
• Sanfilippo Disease Type A

Interventions

Type	Name	Description
GENETIC	SAF-301

Timeline

• Start date: 2013-05-01
• Primary completion: 2017-05-01
• Completion: 2017-06-01
• First posted: 2014-02-03
• Last updated: 2017-06-20

Locations

1 site across 1 country: France