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

Lysogene

Status and phase: Completed; Phase 2; Phase 1

Conditions: Sanfilippo Disease Type A; Mucopolysaccharidosis Type III A

Treatments: Genetic: SAF-301

Study type: Interventional

Funder types: Industry

Identifiers

NCT02053064

P2-SAF-301

Details and patient eligibility

About

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.

Enrollment: 4 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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4 participants in 1 patient group

SAF-301

Experimental group

Treatment:

Genetic: SAF-301