Mucopolysaccharidosis Type III A Clinical Trial
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.
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.
n/a
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02053064 -
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
|
Phase 1/Phase 2 |