Trials / NCT06275620
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Enrolling by invitation Phase 2 Gene therapy
- Sponsor
- Beacon Therapeutics
- Treatment
- AGTC-501 (high dose and standard corticosteroid regimen); AGTC-501 (low dose and standard corticosteroid regimen); AGTC-501 (high dose and modified corticosteroid regimen)
- Gene
- RPGR
- Conditions
- X-Linked Retinitis Pigmentosa
- Ages
- 12 Years to no upper limit
- Enrollment
- 24 participants
- Started
- 2023-11-14
- Main results expected
- 2025-11
- Results posted
- Not yet
- Record last updated
- 2024-10-30
What the study is testing
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
Where it runs
United States (7)
- University of Florida, Jacksonville, Florida
- Bascom Palmer Eye Institute, Miami, Florida
- Boston Children's Hospital, Boston, Massachusetts
- Cincinnati Eye Institute, Cincinnati, Ohio
- Cleveland Clinic, Cleveland, Ohio
- Casey Eye Institute, Portland, Oregon
- Retina Foundation of the Southwest, Dallas, Texas