Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
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.
Mucopolysaccharidosis Type III A
Sanfilippo Disease Type A
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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 of Sanfilippo Type A Syndrome.|
- Tolerance and safety [ Time Frame: during the one year follow-up ] [ Designated as safety issue: Yes ]
- 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)
- 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 ] [ Designated as safety issue: No ]
- Brain MRI
- Neurocognitive/behavioral tests
- Biological markers on blood, urine and CSF
|Study Start Date:||August 2011|
|Estimated Study Completion Date:||January 2014|
|Estimated Primary Completion Date:||January 2014 (Final data collection date for primary outcome measure)|
|Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris|
|Le Kremlin Bicêtre, France, 94275|
|Hôpital Necker, Assistance Publique des Hôpitaux de Paris|
|Paris, France, 75015|