Actively Recruiting
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
Led by Beacon Therapeutics · Updated on 2026-02-19
10
Participants Needed
6
Research Sites
274 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
CONDITIONS
Official Title
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide written informed consent or assent before any study procedures
- Be male aged 12 to 50 years at consent
- Have a documented pathogenic variant in the RPGR gene causing XLRP
- Have a clinical diagnosis of XLRP
- Be in good general health to tolerate surgery and medications
- Be able and willing to follow study instructions and attend visits
- Parents or caregivers must also be able to comply with study requirements if applicable
- Have specific vision levels in both eyes based on ETDRS charts
- Be able to perform all required visual and retinal tests in both eyes
- Have detectable retinal sensitivity and a sub-foveal EZ line confirmed by imaging
You will not qualify if you...
- Have other genetic diseases affecting the retina that interfere with treatment or assessments
- Have history or active oral, genital, or ocular herpes without willingness to use antiviral medication
- Have systemic diseases that prevent gene transfer or surgery if not managed
- Are allergic to corticosteroids or immunosuppressive drugs
- Used certain blood thinners within 7 days before treatment
- Received vaccinations within 60 days before treatment except flu vaccine within 28 days
- Used systemic corticosteroids or immunosuppressants within 3 months before treatment
- Unwilling to use barrier contraception if sexually active during 3 months after treatment
- Have conditions preventing completion of study assessments or suitability
- Participated in other investigational studies recently
- Previously received AAV gene therapy, stem cell therapy, or similar biologics
- Have eye conditions that affect surgery safety or assessment interpretation
- Have significant media opacity or cataracts likely needing surgery soon
- Had intraocular surgery within 90 days before treatment
- Have active eye infections or inflammation
- History of corticosteroid-induced high eye pressure unresponsive to treatment
- Have artificial retinal implants
- Have poor ocular media clarity or pupil dilation for imaging
- History of retinal detachment
- Have very high myopia or pathologic myopia in either eye
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
University of Florida Jacksonville Ophthalmology
Jacksonville, Florida, United States, 32209
Actively Recruiting
2
Bascom Palmer Eye Institute
Miami, Florida, United States, 33136
Not Yet Recruiting
3
Duke Eye Center
Durham, North Carolina, United States, 27710
Actively Recruiting
4
Cincinnati Eye Institute
Cincinnati, Ohio, United States, 45242
Actively Recruiting
5
OHSU Casey Eye Institute
Portland, Oregon, United States, 97239
Actively Recruiting
6
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
Research Team
S
Serva Health Serva Health
CONTACT
A
Amy Christenson
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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