Status:
ACTIVE_NOT_RECRUITING
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
Lead Sponsor:
Beacon Therapeutics
Conditions:
X-Linked Retinitis Pigmentosa
Eligibility:
MALE
12-50 years
Phase:
PHASE2
PHASE3
Brief 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 g...
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 ...
Eligibility Criteria
Inclusion
- General
- 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.
- General
Exclusion
- 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.
- For participants with herpes simplex virus (HSV):
- Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication.
- Have a history of ocular herpes.
- Have active oral or genital herpes or are currently receiving treatment for HSV infection.
- Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
- Have used anti-coagulant agents that may alter coagulation
- 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
- If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following treatment administration.
- Are currently participating or recently participated in any other research
- Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
- Have significant media opacity impacting evaluation of the retina or vitreous. administration.
- Had intraocular surgery within 90 days of study treatment administration.
- Have any active ocular/intraocular infection or inflammation
- Have a history of corticosteroid-induced raised IOP of \>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
- Have any artificial retinal implant or prosthesis.
- Have absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images.
- Have any history of rhegmatogenous retinal detachment.
- 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.
- Have passed the Low Contrast Ora-VNC mobility course at ≤0.35 lux light level in either eye or binocularly at any screening visit.
Key Trial Info
Start Date :
March 14 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2029
Estimated Enrollment :
85 Patients enrolled
Trial Details
Trial ID
NCT04850118
Start Date
March 14 2024
End Date
October 1 2029
Last Update
July 16 2025
Active Locations (24)
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1
Retina Macula Institute of Arizona
Scottsdale, Arizona, United States, 85255
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
3
University of Florida Health Jacksonville, Department of Ophthalmology
Jacksonville, Florida, United States, 32209
4
Bascom Palmer Eye Institute- University of Miami
Miami, Florida, United States, 33136