Trials / NCT00749957
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Completed Phase 1 / Phase 2 Gene therapy
- Sponsor
- Beacon Therapeutics
- Treatment
- rAAV2-CB-hRPE65
- Gene
- RPE65
- Conditions
- Leber Congenital Amaurosis
- Ages
- 6 Years to no upper limit
- Enrollment
- 12 participants
- Started
- 2009-06-17
- Main results expected
- 2014-09-23
- Results posted
- Yes
- Record last updated
- 2017-12-28
What the study is testing
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene.
Funding Source - FDA OOPD
Where it runs
United States (2)
- University of Massachusetts Medical School, Worcester, Massachusetts
- Casey Eye Institue, Oregon Health & Science University, Portland, Oregon