NCT03300453View on ClinicalTrials.gov

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

September 17, 2013

Primary Completion Date

November 27, 2019

Study Completion Date

November 27, 2019

Conditions
Sanfilippo Syndrome B
Interventions
DRUG

rAAV2/5-hNAGLU

one-time brain intraparenchymal gene therapy dose

Trial Locations (1)

94275

Hopitaux Universitaires Paris-Sud, Paris

Sponsors
All Listed Sponsors
collaborator

Venn Life Sciences

OTHER

collaborator

Institut Pasteur

INDUSTRY

lead

UniQure Biopharma B.V.

INDUSTRY