Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
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|ClinicalTrials.gov Identifier: NCT02053064|
Recruitment Status : Completed
First Posted : February 3, 2014
Last Update Posted : June 20, 2017
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|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
- Checking of adverse events [ Time Frame: until 60 months after SAF-301 administration ]
- 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 ]
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 ClinicalTrials.gov identifier (NCT number): NCT02053064
|Hôpitaux Universitaires Paris Sud (Bicêtre)|
|Le Kremlin-Bicêtre, France, 94275|
|Principal Investigator:||Kumaran DEIVA, Dr||AP-HP Hôpital Bicêtre|