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

Phase 2CompletedNCT02053064

LYSOGENE4 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis Type III A Sanfilippo Disease Type A

Interventions

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Checking of adverse events

Time frame: until 60 months after SAF-301 administration

Secondary Outcomes

Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments

Time frame: 5 years after the injection of SAF-301

Information on changes in potential biomarkers of the disease

Time frame: 5 years after the injection of SAF-301

Information to further evaluation of immune response

Time frame: 5 years after the injection of SAF-301

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

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes