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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Opus Genetics, Inc
Study ID
NCT07185256
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • OPGx-BEST1 — GENETIC
    Experimental Genetic Therapy

Study Details

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?

Key Dates

Start date
Sep 25, 2025
Status verified
Mar 2026
Primary completion
Aug 31, 2030
Completion
Aug 31, 2030

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: OPGx-BEST1
    OPGx-BEST1, 1.5E9 vg/eye injected one time subretinally

Primary Outcome Measure

Number of dose-limiting toxicity (DLT) events at the dose tested [ Time Frame: 5 years ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Children's Hospital Los AngelesLos AngelesCalifornia90027
Hillary Schwartz-Gale
[email protected]
323-361-1004
Aaron Nagiel, MD (PRINCIPAL_INVESTIGATOR)
Vitreo Retinal AssociatesGainesvilleFlorida34481
Hailey Ahrens
[email protected]
352-300-4212
Christine Kay, MD (PRINCIPAL_INVESTIGATOR)
Cincinnati Eye InstituteCincinnatiOhio45242
Cincinnati Eye Institute Clinical Research Department
[email protected]
513-569-3688
Robert Sisk, MD (PRINCIPAL_INVESTIGATOR)
Retina Foundation of the SouthwestDallasTexas75231
Kaylie Jones, MS
[email protected]
214-363-3911
Mark Pennesi, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
Children's Hospital Los Angeles· Los Angeles, CAVitreo Retinal Associates· Gainesville, FLCincinnati Eye Institute· Cincinnati, OHRetina Foundation of the Southwest· Dallas, TX

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