NCT01208389View on ClinicalTrials.gov

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

November 30, 2010

Primary Completion Date

October 31, 2027

Study Completion Date

October 31, 2027

Conditions
Leber Congenital Amaurosis
Interventions
BIOLOGICAL

voretigene neparvovec-rzyl

One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector in 300 microliters to the contralateral, previously uninjected eye.

Trial Locations (1)

19104

The Children's Hospital of Philadelphia, Philadelphia

All Listed Sponsors
lead

Spark Therapeutics, Inc.

INDUSTRY

NCT01208389 - Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 | Biotech Hunter | Biotech Hunter