Trials / NCT06467344
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Recruiting now Phase 1 / Phase 2 Gene editing, Gene therapy
- Sponsor
- Ascidian Therapeutics, Inc
- Treatment
- ACDN-01
- Gene
- Not stated
- Conditions
- Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
- Ages
- 12 Years to no upper limit
- Enrollment
- 15 participants
- Started
- 2024-06-11
- Main results expected
- 2030-08-01
- Results posted
- Not yet
- Record last updated
- 2026-10-07
What the study is testing
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
Where it runs
United States (11)
- University of San Francisco, San Francisco, California (terminated)
- Vitreo Retinal Associates, Gainesville, Florida (recruiting now)
- Bascom Palmer Eye Institute, Miami, Florida (recruiting now)
- Wilmer Eye Institute at John Hopkins, Baltimore, Maryland (terminated)
- Massachusetts Eye and Ear, Boston, Massachusetts (recruiting now)
- University of Michigan Kellogg Eye Center, Ann Arbor, Michigan (recruiting now)
- Cincinnati Eye Institute, Cincinnati, Ohio (recruiting now)
- Casey Eye Institute OHSU, Portland, Oregon (recruiting now)
- Children's Hospital of Philadelphia, Philadelphia, Pennsylvania (recruiting now)
- Retina Foundation of the Southwest, Dallas, Texas (recruiting now)
- Retina Consultants of Texas, Houston, Texas (recruiting now)