Date: 2011-03-17
Type of information: Initiation of the trial
phase: 1-2
Announcement: initiation of the trial
Company: Lysogene (France)
Product: SAF-301
Action
mechanism: gene therapy. The adeno-associated viral vector serotype 10 is carrying the human SGSH and SUMF1 cDNAs.
Disease: Sanfilippo disease (Mucopolysaccharidosis type IIIA)
Therapeutic area: Rare diseases - Genetic diseases
Country: France
Trial
details: 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. (NCT01474343)
Latest
news: Lysogene prepares to enter clinical trials with SAF-301, its gene therapy product for the treatment of Sanfilippo disease (or Mucopolysaccharidosis type IIIA). SAF-301 uses a recombinant adeno-associated viral (AAV) serotype 5 as a vehicle to deliver genes encoding SGSH (sulfamidase) and SUMF1 (sulfatase-modifying factor 1) to the brain. This viral vector will be introduced into patient’s brain by stereotaxic surgery.
