OPGx-BEST1 for Best Vitelliform Macular Dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB)
This study is testing a new genetic therapy called OPGx-BEST1 for people with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Researchers want to see if OPGx-BEST1 is safe and how well it works to treat these eye conditions. The therapy involves a single injection into one eye. The study will also help determine the best dose of OPGx-BEST1 for future development. You may be able to join if you are 18 or older and have BVMD or ARB. The study will look at any side effects and how well your vision is doing over 5 years.
- Study design
- This is an open-label (meaning you and your doctors will know what treatment you are receiving), dose-exploring study. It plans to enroll 10 adult participants with ARB or BVMD.
- What's involved
- You will receive a single injection of OPGx-BEST1 into one eye. The study will follow you for 5 years after the injection to monitor for side effects and treatment effectiveness.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 5 years after the injection to assess safety and effectiveness.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
At a glance
Conditions
NCT07185256
Where you'd take part
This study runs at 4 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Children's Hospital Los Angeles
Los Angeles, Californiastudy coordinator listed
Recruiting
Cincinnati Eye Institute
Cincinnati, Ohiostudy coordinator listed
Recruiting
Retina Foundation of the Southwest
Dallas, Texasstudy coordinator listed
Recruiting
Vitreo Retinal Associates
Gainesville, Floridastudy coordinator listed
Not yet recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Number of dose-limiting toxicity (DLT) events at the dose tested5 years
Number of dose-limiting toxicity (DLT) events, defined as any ≥Grade 3 toxicity that occurs within 90 days after IMP administration based on the National Cancer Institute common toxicity criteria
- Number and severity of procedure-related adverse events5 years
Number and severity of adverse events related to the administration of OPGx-BEST1 involving pars plana vitrectomy and subretinal injection under general anesthesia
- Number and severity of adverse events related to OPGx-BEST15 years
Number and severity of adverse events considered related to the study drug, OPGx-BEST1