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

Phase 1

Completed

• Conditions: Mucopolysaccharidosis Type III A; Sanfilippo Disease Type A
• Interventions: Genetic: SAF-301

• Registration Number: NCT02053064
• Lead Sponsor: LYSOGENE

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-05
• Study First Submitted: 2014-01-16
• Study First Submitted QC: 2014-01-31
• Study First Posted: 2014-02-03
• Primary Completion: 2017-05
• Status Verified: 2017-06
• Study Completion: 2017-06
• Last Update Submitted: 2017-06-19
• Last Update Posted: 2017-06-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SAF-301	SAF-301	-

Primary Outcome Measures

Name	Time	Method
Checking of adverse events	until 60 months after SAF-301 administration

Secondary Outcome Measures

Name	Time	Method
Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments	5 years after the injection of SAF-301
Information on changes in potential biomarkers of the disease	5 years after the injection of SAF-301
Information to further evaluation of immune response	5 years after the injection of SAF-301

Trial Locations

Locations (1)

Hôpitaux Universitaires Paris Sud (Bicêtre); 🇫🇷 Le Kremlin-Bicêtre, France