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Evaluating a new treatment for X-Linked Retinitis Pigmentosa

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

Phase2; Phase3 Interventional Beacon Therapeutics · NCT04850118

This study is testing a new injection to see if it can improve vision and is specifically for boys and men with X-Linked Retinitis Pigmentosa caused by certain gene mutations.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment75 (estimated)
Ages12 Years to 50 Years
SexMale
SponsorBeacon Therapeutics Industry-sponsored
Locations24 sites (Scottsdale, Arizona and 23 other locations)
Trial IDNCT04850118 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of a single subretinal injection of AGTC-501, a recombinant adeno-associated virus vector, in male participants with X-Linked Retinitis Pigmentosa (XLRP) caused by RPGR mutations. The study is randomized and controlled, comparing two doses of AGTC-501 against an untreated control group. Approximately 75 eligible male participants aged 12 to 50 will be enrolled and randomized into three groups to assess the treatment's impact on visual function and safety. Participants will be monitored closely throughout the study duration.

Who should consider this trial

Good fit: Ideal candidates are male individuals aged 12 to 50 with a clinical diagnosis of XLRP and specific visual acuity criteria.

Not a fit: Patients who are female or do not have a documented pathogenic variant in the RPGR gene may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve vision and quality of life for patients with XLRP.

How similar studies have performed: Other studies using gene therapy approaches for retinal diseases have shown promising results, indicating potential for success in this novel treatment.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Scottsdale, Arizona and 23 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions X-Linked Retinitis PigmentosaXLRPretinal degenerationRPGRadeno-associated virusAAVgene therapy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.