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NCT07185256 · ClinicalTrials.gov registry record · Phase 1
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
A Phase 1 study of Best Vitelliform Macular Dystrophy and ARB, sponsored by Opus Genetics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 4
- Study locations
NCT07185256: Recruiting Phase 1 study of Best Vitelliform Macular Dystrophy and ARB, sponsored by Opus Genetics.
NCT07185256 is a Phase 1 study of Best Vitelliform Macular Dystrophy and ARB that is actively recruiting participants, run by Opus Genetics. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07185256, a Phase 1 study of Best Vitelliform Macular Dystrophy and ARB, is actively recruiting participants, sponsored by Opus Genetics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 4
- Study locations
Study Summary
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?
Primary Outcome
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
Conditions Studied
Interventions
- GENETIC OPGx-BEST1
Study Locations (4)
California
- Children's Hospital Los Angeles - Los Angeles
Florida
- Vitreo Retinal Associates - Gainesville
Ohio
- Cincinnati Eye Institute - Cincinnati
Texas
- Retina Foundation of the Southwest - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2025-09-25 |
| Est. Completion | 2030-08 |
| Phase | Phase 1 |
What NCT07185256 shows while recruiting
NCT07185256 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 4 conditions, with Best Vitelliform Macular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which OPGx-BEST1 is the first listed.
NCT07185256 reports a single indexed study location in California, Florida, Ohio.
Frequently Asked Questions
What is clinical trial NCT07185256 about?
NCT07185256 is a clinical study titled "Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)". 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 ...
What is the current status of trial NCT07185256?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2025-09-25. Estimated completion is 2030-08.
What conditions does trial NCT07185256 study?
This clinical trial studies the following conditions: Best Vitelliform Macular Dystrophy, ARB, BVMD, Autosomal-Dominant Bestrophinopathy.
What interventions are being tested in trial NCT07185256?
The interventions under investigation include: OPGx-BEST1 (GENETIC).
Who is sponsoring clinical trial NCT07185256?
This trial is sponsored by Opus Genetics, which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07185256 being conducted?
This trial has 4 study locations across California, Florida, Ohio, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT07185256's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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