ACTIVE_NOT_RECRUITING

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

Conditions

X-Linked Retinitis Pigmentosa XLRP retinal degeneration RPGR adeno-associated virus AAV gene therapy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2024-03-14

Study Completion:2029-10

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04850118

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years to 50 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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. 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.	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.

Inclusion Criteria

Exclusion 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.
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.

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.

Contacts and Locations

Principal Investigator

Carrie Reichley

STUDY_DIRECTOR

Beacon Therapeutics

Study Locations (Sites)

Retina Macula Institute of Arizona

Scottsdale, Arizona, 85255

United States

Children's Hospital Los Angeles

Los Angeles, California, 90027

United States

University of Florida Health Jacksonville, Department of Ophthalmology

Jacksonville, Florida, 32209

United States

Bascom Palmer Eye Institute- University of Miami

Miami, Florida, 33136

United States

Midwest Eye Institute (Retina Partners Midwest)

Carmel, Indiana, 46290

United States

Wilmer Eye Institute at Johns Hopkins

Baltimore, Maryland, 21287

United States

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114

United States

Mayo Clinic

Rochester, Minnesota, 55905

United States

Duke Eye Center

Durham, North Carolina, 27710

United States

Cincinnati Eye Institute

Cincinnati, Ohio, 45242

United States

Cole Eye Institute - Cleveland Clinic

Cleveland, Ohio, 44195

United States

Casey Eye Institute, OHSU

Portland, Oregon, 97239

United States

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

Philadelphia, Pennsylvania, 19104

United States

Mid Atlantic Retina

Philadelphia, Pennsylvania, 19107

United States

University of Pittsburgh

Pittsburgh, Pennsylvania, 15219

United States

Retina Consultants of Texas

Bellaire, Texas, 77401

United States

Retina Foundation of the Southwest

Dallas, Texas, 75231

United States

Baylor Eye Institute

Houston, Texas, 77030

United States

Retina Consultants of San Antonio Texas

San Antonio, Texas, 78240

United States

Collaborators and Investigators

Sponsor: Beacon Therapeutics

Carrie Reichley, STUDY_DIRECTOR, Beacon Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-03-14

Study Completion Date2029-10

Study Record Updates

Study Start Date2024-03-14

Study Completion Date2029-10

Terms related to this study

Keywords Provided by Researchers

XLRP
retinal degeneration
RPGR
adeno-associated virus
AAV
gene therapy

Additional Relevant MeSH Terms

X-Linked Retinitis Pigmentosa