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A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Sponsor: Beacon Therapeutics
Summary
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.
Official title: A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa
Key Details
Gender
MALE
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2023-11-14
Completion Date
2029-12
Last Updated
2024-10-30
Healthy Volunteers
No
Conditions
Interventions
AGTC-501 (high dose and standard corticosteroid regimen)
Adeno-associated virus vector expressing a human RPGR gene
AGTC-501 (low dose and standard corticosteroid regimen)
Adeno-associated virus vector expressing a human RPGR gene
AGTC-501 (high dose and modified corticosteroid regimen)
Adeno-associated virus vector expressing a human RPGR gene
Locations (7)
University of Florida
Jacksonville, Florida, United States
Bascom Palmer Eye Institute
Miami, Florida, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Cincinnati Eye Institute
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Casey Eye Institute
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States