Clinical Trial: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment

Brief Summary:

The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the human SGSH and SUMF1 cDNAs for the treatment of Sanfilippo type A syndrome The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.

The primary objective is to assess the tolerance and the safety associated to the proposed treatment through a one-year follow up.

The secondary objective is to collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies.

Four patients will be included in the clinical trial and will be followed during one year.

The enrollment and the follow-up of the patients will take place at Bicêtre Hospital. The Neurosurgery will be performed at Necker-Enfants Malades Hospital.

Safety will be evaluating on clinical, radiological and biological parameters.


Detailed Summary:
Sponsor: LYSOGENE

Current Primary Outcome: Tolerance and safety [ Time Frame: during the one year follow-up ]

Measured by

  • adverse events (by type and severity)
  • clinical parameters (fever, seizure, headache, abnormal somnolence or lethargy, any new neurological symptoms),
  • radiological parameters (on MRI, any sign of bleeding after surgery, any hypersignal on T2 weighted images or diffusion images that are not at the points of injection, and any necrotic area evaluated through T1-weighted and diffusion imaging as well as modification of lipids in spectroscopy)
  • biological parameters (in particular anemia, leucopenia, thrombopenia, liver dysfunction)


Original Primary Outcome: Same as current

Current Secondary Outcome: To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies [ Time Frame: during the one year follow-up ]

  • Brain MRI
  • Neurocognitive/behavioral tests
  • Biological markers on blood, urine and CSF


Original Secondary Outcome: Same as current

Information By: LYSOGENE

Dates:
Date Received: November 10, 2011
Date Started: August 2011
Date Completion:
Last Updated: May 5, 2014
Last Verified: May 2014