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ACTIVE NOT RECRUITING
NCT04850118
PHASE2/PHASE3

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

Sponsor: 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.

Official title: 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

Key Details

Gender

MALE

Age Range

12 Years - 50 Years

Study Type

INTERVENTIONAL

Enrollment

85

Start Date

2024-03-14

Completion Date

2031-10-31

Last Updated

2026-04-07

Healthy Volunteers

No

Interventions

BIOLOGICAL

rAAV2tYF-GRK1-hRPGRco

Adeno-associated virus vector expressing a human RPGR gene

DRUG

Control

Untreated Control Group 3

Locations (25)

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

Cole Eye Institute - Cleveland Clinic

Cleveland, Ohio, United States

Casey Eye Institute, OHSU

Portland, Oregon, United States

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

Philadelphia, Pennsylvania, United States

Mid Atlantic Retina

Philadelphia, Pennsylvania, United States

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Retina Consultants of Texas

Bellaire, Texas, United States

Retina Foundation of the Southwest

Dallas, Texas, United States

Baylor Eye Institute

Houston, Texas, United States

Retina Consultants of San Antonio Texas

San Antonio, Texas, United States

Sydney Eye Hospital

Sydney, New South Wales, Australia

Royal Victorian Eye & Ear Hospital

East Melbourne, Victoria, Australia

McGill University Health Centre - Glen Site

Montreal, Quebec, Canada

Moorfields Eye Hospital

London, United Kingdom

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

London, United Kingdom

Oxford Eye Hospital

Oxford, United Kingdom