Actively Recruiting
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
Led by Opus Genetics, Inc · Updated on 2026-03-24
10
Participants Needed
4
Research Sites
253 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-BEST1. The main questions it aims to answer are: Evaluate the safety and tolerability of drug OPGx-BEST1 in one eye (the treatment eye), for 5 years post-injection, in participants with BVMD or ARB. A second question it aims to answer is identification of the most appropriate dose strength of OPGx-BEST1 for clinical development. Evaluate the efficacy of single injection of OPGx-BEST1 in one eye for 5 years post-injection. What medical problems do participants have when taking drug OPGx-BEST1?
CONDITIONS
Official Title
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide informed consent to study assessments.
- Able and willing to comply with all study assessments for the duration of the study.
- 18 years of age or older.
- ETDRS BCVA for sentinel participant: 20 letters or fewer (Snellen 20/200 or worse).
- ETDRS BCVA for subsequent participants: between 65 and 20 letters inclusive (Snellen 20/50 to 20/200).
- Genetic confirmation of BVMD or ARB with a BEST1 gene variant by certified laboratory.
- Confirmation of one pathogenic or likely pathogenic autosomal-dominant variant in BEST1 for BVMD, or two variants for ARB.
- Clinical diagnosis consistent with advanced BVMD with active subretinal fluid or vitelliform material.
- Clinical diagnosis consistent with ARB.
You will not qualify if you...
- Women of childbearing potential who are pregnant, lactating, or unwilling to use effective contraception from Screening through 1 year post-treatment.
- Men unwilling to use adequate contraception from Screening through 180 days post-treatment.
- Pre-existing eye condition or systemic disease that prevents planned surgery or immunosuppressive treatment.
- History of other bestrophinopathy disorders or conditions interfering with outcome testing.
- Prior gene therapy treatment.
- Presence of active choroidal neovascularization affecting vision or requiring treatment.
- Subretinal fibrosis limiting visual acuity improvement.
- Epiretinal membrane requiring surgery.
- Tube surgery for glaucoma or unstable glaucoma within 4 years.
- Intraocular surgery within 90 days before planned treatment or active inflammation from prior surgery.
- Use of investigational drugs or devices within 90 days before treatment.
- Vaccination within 6 weeks before treatment or planned vaccination within 6 months after.
- Anticoagulant therapy within 2 weeks before treatment.
- Active macular neovascularization.
- Incapable of performing visual function testing for reasons other than poor vision.
- Contraindication to the required steroid regimen.
- Any ocular, medical, or psychological condition preventing study completion or making participation unsafe.
- Known allergy to components of the study drug.
- Known HIV, hepatitis B or C, or herpes simplex virus infection.
- Employment or close relation to the study sponsor or site staff.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Vitreo Retinal Associates
Gainesville, Florida, United States, 34481
Not Yet Recruiting
3
Cincinnati Eye Institute
Cincinnati, Ohio, United States, 45242
Actively Recruiting
4
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
Research Team
S
Study Director
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here