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A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
Sponsor: Beacon Therapeutics
Summary
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.
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 Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
Key Details
Gender
MALE
Age Range
8 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
14
Start Date
2021-04-13
Completion Date
2027-02
Last Updated
2024-08-12
Healthy Volunteers
No
Conditions
Interventions
rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Locations (6)
University of Florida
Jacksonville, Florida, United States
Boston Children's Hosptial
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