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Also known as OCU400 Low Dose, OCU400 Med Dose, OCU400 Second Eye Dosing, Sub-Retinal Administration of OCU400-301
OCU400 · 2 trials · 2 indications
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)
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 |
|---|---|---|
| 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 |
| 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 |
|---|---|---|
| Sub-Retinal Administration of OCU400-301 | GENETIC | Sub-Retinal Administration of OCU400-301 |
| 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 |
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...
OCU400 Low Dose is an investigational small molecule being developed for the treatment of Retinitis Pigmentosa, a rare inherited retinal disease. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is also being studied in patients with Leber Congenital Amaurosis.
OCU400 Low Dose targets the NR2E3 receptor, a nuclear receptor involved in retinal cell function and survival. By modulating this target, the drug aims to address the underlying genetic causes of Retinitis Pigmentosa and potentially slow or halt disease progression.
OCU400 Low Dose is being developed by Ocugen, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol OCGN. Ocugen is conducting clinical research on this drug candidate for rare retinal diseases.
OCU400 Low Dose is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 1 trial is active but not recruiting participants, and the drug has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA.
OCU400 Low Dose is being evaluated in a single Phase 1 clinical trial with the identifier NCT05203939. This trial is titled 'Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis' and is enrolling 22 participants in the United States.
OCU400 Low Dose is a specific dosage form of the drug candidate OCU400, which is being developed by Ocugen. The clinical trial NCT05203939 evaluates OCU400 in patients with Retinitis Pigmentosa and Leber Congenital Amaurosis, and the low dose formulation is part of this broader development program.