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
180 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
X-linked retinitis pigmentosa (XLRP) is an inherited eye condition that affects the retina, leading to night blindness and gradual vision loss. This Phase 2 study evaluates the investigational drug laruparetigene zovaparvovec (AGTC-501) to see if it is safe and whether it can preserve or improve vision and other symptoms in male participants with XLRP. The trial focuses on males aged 12 to 50 years who have a genetic diagnosis of XLRP related to the RPGR gene. Participants will undergo surgery called pars plana vitrectomy to receive laruparetigene zovaparvovec by injection under the retina in both eyes. The first treated eye is dosed on Day 1, and the second eye is treated either 30 to 60 days later or sooner (7 to 14 days) depending on safety reviews. The study drug is a biological treatment delivered through a subretinal injection. Researchers will monitor participants for up to 5 years after treatment. During the study, participants will have regular eye exams and vision tests, including measures of visual acuity, light sensitivity, and retinal structure. Safety will be closely monitored by tracking any serious or significant side effects over the first year and longer term. Questionnaires and virtual reality tests will assess daily vision-related functions. The main outcome is the number of participants experiencing severe treatment-related adverse events within 12 months. The total participation time may last several years with multiple follow-up visits.
CONDITIONS
Brief 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 inclusive
- Have a documented pathogenic or likely pathogenic RPGR gene variant
- Have a clinical diagnosis of X-linked retinitis pigmentosa
- Be in good health to undergo subretinal surgery and related medications
- Be able and willing to follow study instructions and attend visits
- Have a parent or caregiver able to support study participation, if applicable
- Have best corrected visual acuity between 34 and 78 ETDRS letters in each eye
- Have low-luminance visual acuity of 64 letters or less in both eyes
- Have a low-luminance deficit greater than 10 letters
- Be able to perform visual and retinal function tests reliably in both eyes
- Have detectable mean macular sensitivity between 1 and 12 dB by microperimetry in both eyes
- Have a detectable sub-foveal ellipsoid zone line by optical coherence tomography in both eyes
You will not qualify if you...
- Have other disease-causing mutations that may affect treatment or assessments
- Have history of oral or genital herpes and cannot use antiviral medication
- Have history of ocular herpes or active herpes infection
- Have systemic diseases causing immunosuppression or active infection
- Have allergy or sensitivity to corticosteroids or immunosuppressive drugs
- Have used anticoagulant agents (excluding ibuprofen or aspirin) within 7 days prior to treatment
- Have received vaccinations (except flu vaccine beyond 28 days) within 60 days prior to treatment
- Have used systemic corticosteroids or immunosuppressants within 3 months prior to treatment
- Are unwilling to use barrier contraception if sexually active
- Have any condition making study completion unlikely or unsuitable
- Have recently participated in other investigational therapy studies within 90 days or 10 drug half-lives
- Have received any gene therapy, stem cell, or similar biologic treatments previously
- Have eye conditions preventing surgery or affecting results (e.g., corneal opacities, diabetic retinopathy, glaucoma)
- Have significant media opacity or cataracts likely requiring surgery within 3 months
- Have had eye surgery within 90 days before treatment
- Have active ocular infection or inflammation
- Have history of corticosteroid-induced high intraocular pressure
- Have artificial retinal implants
- Have inadequate ocular media clarity or pupil dilation
- Have history of retinal detachment
- Have severe myopia exceeding -10 diopters or pathologic myopia in either eye
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Day 1 to up to 60 days
Participants undergo a pars plana vitrectomy and receive a central subretinal administration of laruparetigene zovaparvovec in their first treated eye. The second eye is treated between 7 and 60 days after the first surgery, depending on safety reviews.
1 to 2 surgical visits
Duration - Up to 12 months
Participants are monitored for safety and vision outcomes following surgery and treatment in both eyes.
Multiple visits during the first year for assessments
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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