A Phase 1b/2a, Open-Label, Dose-Exploration Basket Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients With Either Autosomal-Dominant BEST1 Disease (Best Vitelliform Macular Dystrophy [BVMD]) or Autosomal-Recessive Bestrophinopathy (ARB) (BIRD-1)

NCT07185256 Phase 1/Phase 2 RECRUITING

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?

Details

Lead sponsor	Opus Genetics, Inc
Phase	Phase 1/Phase 2
Status	RECRUITING
Enrolment	10
Start date	2025-09-25
Completion	2030-08

Conditions

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

Interventions

• OPGx-BEST1

Primary outcomes

• Number of dose-limiting toxicity (DLT) events at the dose tested — 5 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 events — 5 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-BEST1 — 5 years
  Number and severity of adverse events considered related to the study drug, OPGx-BEST1

Countries

United States