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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05203939 | Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis | PHASE1 | ACTIVE NOT_RECRUITING | 22 | — | — | Jan 24, 2022 | Mar 1, 2027 | Aug 6, 2025 | 7 | United States |
Counts, frequencies and percentages of SDAEs. SDAE is a primary adverse event of interest and defined as AEs and SAEs that are direct subjects to the Study Drug only.
Counts, frequencies and percentages TEAEs. TEAEs are defined as an event that was not present prior to administration of the dose of study drug and present after the dose, or if it represents the exacerbation of an event that was present prior to the dose.
Counts, frequencies and percentages of SAEs including Resulted in Death, Life-threatening, Hospitalization, Disabling/incapacitating, Congenital anomaly or birth defect and medically significant AEs ( AE that did not meet any of the above criteria but could have jeopardized the subject and might have required medical or surgical intervention to prevent one of the outcomes listed above).
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (Low Dose) | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup |
| Cohort 2 (Mid Dose) | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup |
| Cohort 3 (High Dose) | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup |
| Pediatric Arm | EXPERIMENTAL | Pediatric subjects will receive the medium dose concentration |
| Phase 2 (High and Medium Dose) | EXPERIMENTAL | Following DSMB confirmation, adult RP subjects with Biallelic autosomal recessive NR2E3 mutations, autosomal dominant NR2E3 mutations, Autosomal dominant RHO mutations will receive a high dose concentration of OCU400 or LCA patients with CEP290 mutation will receive a medium dose concentration of OCU400. |
| Adult Arm | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup will receive a high dose concentration of OCU400 and LCA patients with CEP290 will receive a medium dose concentration of OCU400 |
| Second Eye Dosing | EXPERIMENTAL | Eligible RP participants will be dosed in the untreated fellow eye with a therapeutic dose used in Phase 3 study of OCU400 (1.0x10E11vg/mL in 250 μl ) and will be followed for an additional 48 weeks. |
| Natural History Study (OCU400-104) | NO_INTERVENTION | A Prospective and Retrospective Natural History Study of RP and LCA: This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects. The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with earliest timepoint on or after the date of their diagnosis of RP or LCA. Subjects will be seen up to a total of four times during the 12 months of the Observational Period, at baseline, 3 months, 6 months and 12 months. A total of up to 100 subjects will be enrolled in the study, including: Approximately 76 newly enrolled subjects consisting of 50 adult RP subjects, 6 adult LCA subjects, 20 pediatric RP/LCA subjects. Up to 24 subjects that reconsent from the OCU400-101 study (subjects from OCU400-101 will provide data on their untreated eye) |
| Name | Type | Description |
|---|---|---|
| OCU400 Low Dose | DRUG | subretinal injection of up to 1.66×10E10 vg/mL |
| OCU400 Med Dose | DRUG | subretinal injection of up to 3.33×10E10 vg/mL |
| OCU400 High Dose | DRUG | subretinal injection of up to 1.66×10E11 vg/mL |
| OCU400 Second Eye Dosing | DRUG | subretinal injection of 1.0x10E11vg/mL in 250 μl |
Diagnosis and main criteria for inclusion: Subjects meeting all inclusion criteria and none of the exclusion criteria are eligible for study participation. Inclusion Criteria for Adult RP: 1. Males or females ≥ 18 years of age at the time of informed consent. 2. Confirmed genetic diagnosis of bia...