Clinical Trial: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
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 Administra
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 Summary:
Sponsor: LYSOGENE
Current Primary Outcome: Checking of adverse events [ Time Frame: until 60 months after SAF-301 administration ]
Original Primary Outcome: Checking of adverse events [ Time Frame: until 32 months after SAF-301 administration ]
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome:
- Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments [ Time Frame: 2 years after the injection of SAF-301 ]
- Information on changes in potential biomarkers of the disease [ Time Frame: 2 years after the injection of SAF-301 ]
- Information to further evaluation of immune response [ Time Frame: 2 years after the injection of SAF-301 ]
Information By: LYSOGENE
Dates:
Date Received: January 16, 2014
Date Started: May 2013
Date Completion: June 2017
Last Updated: January 17, 2017
Last Verified: January 2017