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ACTIVE_NOT_RECRUITINGPHASE2/PHASE3

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

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

NCT04850118•Beacon Therapeutics

View on ClinicalTrials.gov

Summary

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.

Detailed Description

This study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1 or Dose 2 will be administered in participants in 2 treatment groups while participants in the untreated control group will be followed and evaluated, after which they will be evaluated to determine eligibility to receive treatment with AGTC-501 Dose 2. Approximately 75 eligible male participants between 12 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups.

Eligibility

Age

12 - 50 years

Sex

MALE

Healthy Volunteers

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.
•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.
+1 more criteria

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
+10 more criteria

Locations (24)

Retina Macula Institute of Arizona

Scottsdale, Arizona, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

University of Florida Health Jacksonville, Department of Ophthalmology

Jacksonville, Florida, United States

Bascom Palmer Eye Institute- University of Miami

Miami, Florida, United States

Midwest Eye Institute (Retina Partners Midwest)

Carmel, Indiana, United States

Wilmer Eye Institute at Johns Hopkins

Baltimore, Maryland, United States

Ophthalmic Consultants of Boston

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

Duke Eye Center

Durham, North Carolina, United States

Cincinnati Eye Institute

Cincinnati, Ohio, United States

Key Dates

Start Date

March 14, 2024

Primary Completion Date

August 1, 2025

Completion Date

October 1, 2029

Last Updated

July 16, 2025

Enrollment

ACTUAL participants

Conditions

X-Linked Retinitis Pigmentosa

Interventions

rAAV2tYF-GRK1-hRPGRco

BIOLOGICAL

Control

DRUG

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

NCT07174726

X-Linked Retinitis Pigmentosa (XLRP)

View study

RECRUITINGPHASE1/PHASE2

Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

NCT06492850

X-Linked Retinitis Pigmentosa (XLRP)

View study

ACTIVE_NOT_RECRUITINGPHASE2

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 16, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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