Trials / NCT06333249
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
Underway, not enrolling Phase 2 Gene therapy
- Sponsor
- Beacon Therapeutics
- Treatment
- rAAV2tYF-GRK1-RPGR
- Gene
- RPGR
- Conditions
- X-Linked Retinitis Pigmentosa
- Ages
- 8 Years to 50 Years
- Enrollment
- 14 participants
- Started
- 2021-04-13
- Main results expected
- 2023-04-11
- Results posted
- Not yet
- Record last updated
- 2024-08-12
What the study is testing
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
Where it runs
United States (6)
- University of Florida, Jacksonville, Florida
- Boston Children's Hosptial, Boston, Massachusetts
- Cincinnati Eye Institute, Cincinnati, Ohio
- Cleveland Clinic, Cleveland, Ohio
- Casey Eye Institute, Portland, Oregon
- Retina Foundation of the Southwest, Dallas, Texas