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

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Disease Information

Descriptive Information
Brief Title † Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Official Title † 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.
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
Study Phase Phase 1/Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Checking of adverse events
Secondary Outcome Measure † Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments
Condition † Mucopolysaccharidosis Type III A Sanfilippo Disease Type A
Intervention † GeneticSAF-301
Study Arms / Comparison Groups SAF-301
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Genetic
Estimated Enrollment † 4
Start Date † May 2013
Completion Date June 2017
Primary Completion Date May 2017
Eligibility Criteria † 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.
Gender All
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † France
Administrative Information
NCT ID † NCT02053064
Organization ID P2-SAF-301
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † LYSOGENE
Collaborators ††
Investigators † Principal Investigator: Kumaran DEIVA, Dr, AP-HP Hôpital Bicêtre
Information Provided By
Verification Date January 2017
First Received Date † January 16, 2014
Last Updated Date January 17, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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