NCT01474343 - Intracerebral Gene Therapy for Sanfilippo Type A Syndrome | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

August 31, 2011

Primary Completion Date

May 31, 2013

Study Completion Date

May 31, 2013

Conditions

Mucopolysaccharidosis Type III ASanfilippo Disease Type A

Interventions

GENETIC

SAF-301

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.

Trial Locations (2)

75015

Hôpital Necker, Assistance Publique des Hôpitaux de Paris, Paris

94275

Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris, Le Kremlin-Bicêtre