๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-04-06

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

๐Ÿ”ต Active, Not Recruiting Phase2 / Phase3 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: Beacon Therapeutics

๐Ÿฉบ 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

๐Ÿงช Interventions in This Study

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ 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,

๐Ÿ’ฐ 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.

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—