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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06388200 | A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa | PHASE3 | ACTIVE NOT_RECRUITING | 140 | — | — | Jun 18, 2024 | Feb 12, 2027 | Mar 5, 2026 | 17 | United States, Canada |
Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
| Arm | Type | Description |
|---|---|---|
| RHO Arm | EXPERIMENTAL | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye |
| Gene Agnostic Arm | EXPERIMENTAL | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye |
| Control for RHO Arm | NO_INTERVENTION | Will not receive any active study intervention |
| Control for Gene Agnostic Arm | NO_INTERVENTION | Will not receive any active study intervention |
| Name | Type | Description |
|---|---|---|
| Sub-Retinal Administration of OCU400-301 | GENETIC | Sub-Retinal Administration of OCU400-301 |
Inclusion Criteria: 1. Males or females ≥ 3 years of age 2. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP 3. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2...