Trials / NCT03872479
Single Ascending Dose Study in Participants With LCA10
Status unknown Phase 1 / Phase 2 Gene editing, Gene therapy
- Sponsor
- Editas Medicine, Inc.
- Treatment
- EDIT-101
- Gene
- CEP290
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
- Ages
- 3 Years to no upper limit
- Enrollment
- 34 participants
- Started
- 2019-09-26
- Main results expected
- 2025-05-23
- Results posted
- Not yet
- Record last updated
- 2022-12-05
What the study is testing
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26").
Where it runs
United States (5)
- Bascom Palmer Eye Institute, Miami, Florida
- Massachusetts Eye and Ear Infirmary, Boston, Massachusetts
- W.K. Kellogg Eye Center - University of Michigan, Ann Arbor, Michigan
- Casey Eye Institute - OSHU, Portland, Oregon
- University of Pennsylvania, Philadelphia, Pennsylvania