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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02053064
Recruitment Status : Completed
First Posted : February 3, 2014
Last Update Posted : June 20, 2017
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis Type III A Sanfilippo Disease Type A Genetic: SAF-301 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Study Start Date : May 2013
Actual Primary Completion Date : May 2017
Actual Study Completion Date : June 2017

Arm Intervention/treatment
Experimental: SAF-301 Genetic: SAF-301

Primary Outcome Measures :
  1. Checking of adverse events [ Time Frame: until 60 months after SAF-301 administration ]

Secondary Outcome Measures :
  1. 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 ]
  2. Information on changes in potential biomarkers of the disease [ Time Frame: 5 years after the injection of SAF-301 ]
  3. Information to further evaluation of immune response [ Time Frame: 5 years after the injection of SAF-301 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02053064

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Hôpitaux Universitaires Paris Sud (Bicêtre)
Le Kremlin-Bicêtre, France, 94275
Sponsors and Collaborators
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Principal Investigator: Kumaran DEIVA, Dr AP-HP Hôpital Bicêtre
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Responsible Party: LYSOGENE Identifier: NCT02053064    
Other Study ID Numbers: P2-SAF-301
First Posted: February 3, 2014    Key Record Dates
Last Update Posted: June 20, 2017
Last Verified: June 2017
Keywords provided by LYSOGENE:
Neurodegenerative diseases
Nervous systems diseases
Genetic diseases, Inborn
Metabolic diseases
Gene therapy
Adeno Associated Virus (AAV)
Additional relevant MeSH terms:
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Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases