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

NCT07185256 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-03-24

No results posted yet for this 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?

Conditions

  • ARB
  • BVMD
  • Autosomal-Dominant Bestrophinopathy
  • Best Vitelliform Macular Dystrophy

Interventions

GENETIC

OPGx-BEST1

Experimental Genetic Therapy

Sponsors & Collaborators

  • Opus Genetics, Inc

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-09-25
Primary Completion
2030-08-31
Completion
2030-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07185256 on ClinicalTrials.gov