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Study identifier: NCT07185256 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

Condition: ARB · BVMD · Autosomal-Dominant Bestrophinopathy  ·  Sponsor: Opus Genetics, Inc

PhasePhase 1/Phase 2
Planned participants10
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

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?

This description comes directly from the study's public registry record.

Talk to the study team

Study Director  ·  984-884-6030  ·  ct.gov_inquiries@opusgtx.com

Always discuss trial participation with your own doctor first.

Locations (4)

Children's Hospital Los AngelesLos Angeles, California, United StatesRecruiting
Vitreo Retinal AssociatesGainesville, Florida, United StatesNot Yet Recruiting
Cincinnati Eye InstituteCincinnati, Ohio, United StatesRecruiting
Retina Foundation of the SouthwestDallas, Texas, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT07185256