Safety Study in Subjects With Leber Congenital Amaurosis

PHASE1CompletedINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

September 30, 2007

Primary Completion Date

March 20, 2018

Study Completion Date

March 20, 2018

Conditions
Leber Congenital Amaurosis
Interventions
BIOLOGICAL

voretigene neparvovec-rzyl

Subjects will be dosed unilaterally (one eye) beginning with the lowest dose. Subjects will be injected with AAV2-hRPE65v2 by means of a subretinal injection. Dose escalation to the next cohort will be dependent on assessment of the safety data by the DSMB out to at least 4 weeks following the injection. Because there is a delay between time of delivery of AAV2 and the peak transgene expression there will be a delay of six weeks between all subjects.

Trial Locations (1)

19104

The Children's Hospital of Philadelphia, Philadelphia

All Listed Sponsors
lead

Spark Therapeutics, Inc.

INDUSTRY

NCT00516477 - Safety Study in Subjects With Leber Congenital Amaurosis | Biotech Hunter | Biotech Hunter