Actively Recruiting
Phase 2 Study to Evaluate Safety of Laruparetigene Zovaparvovec Gene Therapy Given in Both Eyes for Males 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
X-Linked Retinitis Pigmentosa (XLRP) is a genetic eye disease that affects the retina, leading to night blindness and gradual vision loss. This Phase 2 study aims to evaluate if the investigational drug laruparetigene zovaparvovec (AGTC-501), given in both eyes, is safe and can help preserve or improve vision and other symptoms of XLRP. The study focuses on male participants aged 12 to 50 years with documented mutations in the RPGR gene causing XLRP. Participants will receive the investigational drug through subretinal injections in both eyes. The dose of AGTC-501 is administered bilaterally, and the treatment is designed to address the genetic cause of XLRP. The study does not include a placebo or comparator group as it is an open-label trial. During the study, participants will undergo various eye exams including visual acuity tests and retinal imaging to assess vision and retinal function. Researchers will monitor safety by tracking the number and percentage of participants experiencing serious or severe treatment-related side effects over 12 months. Participants will be followed closely with study visits and assessments for one year after treatment to evaluate the drug's safety and its effects on vision.
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
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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
A
Amy Christenson
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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