Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07185256 | Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) | PHASE1 | RECRUITING | 10 | — | — | Sep 25, 2025 | Aug 1, 2030 | Mar 24, 2026 | 4 | United States |
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 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 considered related to the study drug, OPGx-BEST1
| Arm | Type | Description |
|---|---|---|
| OPGx-BEST1 | EXPERIMENTAL | OPGx-BEST1, 1.5E9 vg/eye injected one time subretinally |
| Name | Type | Description |
|---|---|---|
| OPGx-BEST1 | GENETIC | Experimental Genetic Therapy |
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 ...