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

Who is this study for? Patients with X-Linked Retinitis Pigmentosa
What treatments are being studied? rAAV2tYF-GRK1-hRPGRco
Status: Active_not_recruiting
Location: See all (24) locations...
Intervention Type: Biological, Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 12
Maximum Age: 50
Healthy Volunteers: f
View:

• 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.

• Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable).

• Be male (XY chromosome) and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene.

• Have a clinical diagnosis of XLRP.

• 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):

• Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200)

• Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye

• 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.

• Have an LLD of \> 10 letters in the study eye

⁃ 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.

⁃ 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.

Locations
United States
Arizona
Retina Macula Institute of Arizona
Scottsdale
California
Children's Hospital Los Angeles
Los Angeles
Florida
University of Florida Health Jacksonville, Department of Ophthalmology
Jacksonville
Bascom Palmer Eye Institute- University of Miami
Miami
Indiana
Midwest Eye Institute (Retina Partners Midwest)
Carmel
Massachusetts
Ophthalmic Consultants of Boston
Boston
Maryland
Wilmer Eye Institute at Johns Hopkins
Baltimore
Minnesota
Mayo Clinic
Rochester
North Carolina
Duke Eye Center
Durham
Ohio
Cincinnati Eye Institute
Cincinnati
Cole Eye Institute - Cleveland Clinic
Cleveland
Oregon
Casey Eye Institute, OHSU
Portland
Pennsylvania
Mid Atlantic Retina
Philadelphia
The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine
Philadelphia
University of Pittsburgh
Pittsburgh
Texas
Retina Consultants of Texas
Bellaire
Retina Foundation of the Southwest
Dallas
Baylor Eye Institute
Houston
Retina Consultants of San Antonio Texas
San Antonio
Other Locations
Australia
Royal Victorian Eye & Ear Hospital
East Melbourne
Sydney Eye Hospital
Sydney
United Kingdom
Moorfields Eye Hospital
London
The Retina Clinic London, Institute of Ophthalmology, University College London
London
Oxford Eye Hospital
Oxford
Time Frame
Start Date: 2024-03-14
Completion Date: 2029-10
Participants
Target number of participants: 85
Treatments
Active_comparator: Group 1: Dose
Male participants 12-50 years of age treated by subretinal injection with the of AGTC-501
Active_comparator: Group 2: Dose
Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501
Other: Group 3: Control
Male participants 12-50 years of age in the untreated control group. Participants in the control group will be followed for a minimum of 24 months. After all participants have reached Month 12, participants in the control group will be given the option to receive the study drug in the fellow eye, if eligible.
Sponsors
Leads: Beacon Therapeutics

This content was sourced from clinicaltrials.gov

Similar Clinical Trials