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Gene therapy offers hope for rare Blindness-Causing eye disease

NCT ID NCT04850118

Disease control Sponsor: Beacon Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a gene therapy called AGTC-501 in males aged 12 to 50 with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. Participants receive one of two doses of the therapy or no treatment, and researchers measure changes in vision under low light over time. The goal is to see if the treatment can slow or improve vision loss safely.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

85 people

The number who actually took part.

Started

Mar 2024

Expected to finish

Oct 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 50 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

General Inclusion Criteria: 1. Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent. 2. Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable). 3. Be male (XY chromosome) and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene. 4. Have a clinical diagnosis of XLRP. 5. Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend study visits for the duration of the study. Ocular Inclusion Criteria (Study Eye): 6. Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200) 7. Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye 8. Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability, and fixation, in the study eye per the Investigator's discretion. 9. Have an LLD of \> 10 letters in the study eye 10. Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, between 1-12 decibels (dB) in the study eye, as determined by the Investigator and confirmed by the CRC with fixation loss ≤20% at each screening visit. 11. Have a detectable sub-foveal EZ line in the study eye as assessed by spectral domain-optical coherence tomography (SD-OCT) and confirmed by the CRC. General Exclusion Criteria: 1. Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel, that in the opinion of the Investigator would interfere with the potential therapeutic effect of the study agent or the quality of the assessments. 2. For participants with herpes simplex virus (HSV): 1. Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication. 2. Have a history of ocular herpes. 3. Have active oral or genital herpes or are currently receiving treatment for HSV infection. 3. Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications. 4. Have used anti-coagulant agents that may alter coagulation 5. Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening. Corticosteroids used on an as-needed basis administered by insufflation, inhalation or local administration to the skin 6. If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following treatment administration. 7. Are currently participating or recently participated in any other research 8. Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics. 9. Have significant media opacity impacting evaluation of the retina or vitreous. administration. 10. Had intraocular surgery within 90 days of study treatment administration. 11. Have any active ocular/intraocular infection or inflammation 12. Have a history of corticosteroid-induced raised IOP of \>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy. 13. Have any artificial retinal implant or prosthesis. 14. Have absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images. 15. Have any history of rhegmatogenous retinal detachment. 16. Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of \>30 mm if the Principal Investigator \[PI\] deems it appropriate to measure) or presence of pathologic myopia in the study eye. 17. Have passed the Low Contrast Ora-VNC mobility course at ≤0.35 lux light level in either eye or binocularly at any screening visit.

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Conditions

The condition(s) this trial relates to.

retinal degeneration retinitis pigmentosa

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

X-linked retinitis pigmentosa

Contacts and locations

Locations

  • Bascom Palmer Eye Institute- University of Miami

    Miami, Florida, 33136, United States

  • Baylor Eye Institute

    Houston, Texas, 77030, United States

  • Casey Eye Institute, OHSU

    Portland, Oregon, 97239, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242, United States

  • Cole Eye Institute - Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Duke Eye Center

    Durham, North Carolina, 27710, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • McGill University Health Centre - Glen Site

    Montreal, Quebec, H4A3J1, Canada

  • Mid Atlantic Retina

    Philadelphia, Pennsylvania, 19107, United States

  • Midwest Eye Institute (Retina Partners Midwest)

    Carmel, Indiana, 46290, United States

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114, United States

  • Oxford Eye Hospital

    Oxford, OX39DU, United Kingdom

  • Retina Consultants of San Antonio Texas

    San Antonio, Texas, 78240, United States

  • Retina Consultants of Texas

    Bellaire, Texas, 77401, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231, United States

  • Retina Macula Institute of Arizona

    Scottsdale, Arizona, 85255, United States

  • Royal Victorian Eye & Ear Hospital

    East Melbourne, Victoria, 3002, Australia

  • Sydney Eye Hospital

    Sydney, New South Wales, 2000, Australia

  • The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine

    Philadelphia, Pennsylvania, 19104, United States

  • The Retina Clinic London, Institute of Ophthalmology, University College London

    London, W1G7LB, United Kingdom

  • University of Florida Health Jacksonville, Department of Ophthalmology

    Jacksonville, Florida, 32209, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15219, United States

  • Wilmer Eye Institute at Johns Hopkins

    Baltimore, Maryland, 21287, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.