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)
Opus Genetics, Inc
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?
Description
This is a Phase 1b/2a, open-label, dose-exploring, safety and tolerability basket study of a single subretinal injection of OPGx-BEST1 in one eye of adult participants with ARB or BVMD due to BEST1 mutations. OPGx-BEST1 has been bioengineered to include a transgene expression cassette with a codon-optimized, full-length hBEST1 gene under the control of an RPE-specific promoter. The vector genome contains inverted terminal repeats flanking an expression cassette containing the VMD2 promoter, full-length BEST1 ORF followed by SV 40 and bGH polyadenylation signal sequences, and lastly includes a…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Individuals who meet all the following criteria will be eligible to participate in the study: 1. Provide informed consent to study assessments. 2. Able and willing to comply with all study assessments for the duration of the study. 3. ≥18 years old. 4. ETDRS BCVA measured with standard testing distances: 1. For the sentinel participant in each cohort, ≤20 letters (Snellen equivalent of 20/200 \[1.30 logMAR\] or worse) 2. For subsequent participants in the same cohort, 65 to 20 letters inclusive (Snellen equivalent of 20/50 \[0.40 logMAR\] to 20/200 \[1.30 logMAR\])…
Interventions
- GeneticOPGx-BEST1
Experimental Genetic Therapy
Locations (4)
- Children's Hospital Los AngelesLos Angeles, California
- Vitreo Retinal AssociatesGainesville, Florida
- Cincinnati Eye InstituteCincinnati, Ohio
- Retina Foundation of the SouthwestDallas, Texas