A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP (NCT04850118)
A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa
๐ฉบ Plain-English Summary (8th-Grade Level)
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.
Who can join: Review the eligibility criteria below.
Location: Retina Macula Institute of Arizona โ Scottsdale, Arizona
Age range: 12 Years to 50 Years
๐งช Interventions in This Study
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria โ
๐ All Participating Trial Locations (25 Sites)
- Retina Macula Institute of Arizona โ Scottsdale, Arizona
- Children's Hospital Los Angeles โ Los Angeles, California
- University of Florida Health Jacksonville, Department of Ophthalmology โ Jacksonville, Florida
- Bascom Palmer Eye Institute- University of Miami โ Miami, Florida
- Midwest Eye Institute (Retina Partners Midwest) โ Carmel, Indiana
- Wilmer Eye Institute at Johns Hopkins โ Baltimore, Maryland
- Ophthalmic Consultants of Boston โ Boston, Massachusetts
- Mayo Clinic โ Rochester, Minnesota
- Duke Eye Center โ Durham, North Carolina
- Cincinnati Eye Institute โ Cincinnati, Ohio
- Cole Eye Institute - Cleveland Clinic โ Cleveland, Ohio
- Casey Eye Institute, OHSU โ Portland, Oregon
- The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine โ Philadelphia, Pennsylvania
- Mid Atlantic Retina โ Philadelphia, Pennsylvania
- University of Pittsburgh โ Pittsburgh, Pennsylvania
- Retina Consultants of Texas โ Bellaire, Texas
- Retina Foundation of the Southwest โ Dallas, Texas
- Baylor Eye Institute โ Houston, Texas
- Retina Consultants of San Antonio Texas โ San Antonio, Texas
- Sydney Eye Hospital โ Sydney, New South Wales
- Royal Victorian Eye & Ear Hospital โ East Melbourne, Victoria
- McGill University Health Centre - Glen Site โ Montreal, Quebec
- Moorfields Eye Hospital โ London,
- The Retina Clinic London, Institute of Ophthalmology, University College London โ London,
- Oxford Eye Hospital โ Oxford,
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in X-Linked Retinitis Pigmentosa
- Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations โ Durham, North Carolina
- Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene โ La Jolla, California
- A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) โ Boston, Massachusetts
- A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) โ Jacksonville, Florida
- 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) โ Aurora, Colorado
Explore by condition: All X-Linked Retinitis Pigmentosa clinical trials
๐ก How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.