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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-05-31

Study Completion Date

2017-06-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Mucopolysaccharidosis Type III A Sanfilippo Disease Type A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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SAF-301

Group Type EXPERIMENTAL

SAF-301

Intervention Type GENETIC

Interventions

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SAF-301

Intervention Type GENETIC

Eligibility Criteria

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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.