Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Retinitis Pigmentosa, Leber Congenital Amaurosis
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring NR2E3, Rhodopsin, Enhanced S-cone syndrome, Cep290
Eligibility Criteria
Diagnosis and main criteria for inclusion:
Subjects meeting all inclusion criteria and none of the exclusion criteria are eligible for study participation.
Inclusion Criteria:
Males or females ≥18 years of age at the time of informed consent.
Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations for Subgroup 1, autosomal dominant NR2E3 mutation for Subgroup 2 or autosomal dominant RHO mutations for Subgroup 3.
For the sentinel subject of Cohort 1-3, BCVA ≤ 20/160 in study eye or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
For non-sentinel subject, BCVA ≤ 20/50 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
Able to perform a multi-luminance mobility testing (MLMT) using study eye, but unable to pass the MLMT at 1 lux, the lowest luminance level tested.
Subject eligibility for NR2E3 subgroups who pass the MLMT at 1 lux may be assessed on a case-by-case basis by Ocugen following review of results of screening assessments.
Exclusion Criteria:
Subject lacks evidence of outer nuclear layer, i.e., containing the nuclei of the retinal photoreceptors as determined by spectral-domain optical coherence tomography (SD-OCT).
Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator, i.e., inability to fixate, high myopia ≥10 diopters, glaucoma, medium haze, other retinal pathologies, and > 3-fold elevation of liver enzymes or > 2-fold elevation of serum creatinine, etc.
Previous treatment with a gene-therapy or cell therapy product.
Previous treatment with any investigational drug or device within one year.
Any contraindications for subretinal injection.
Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period. (Section 8.3.2)
Any medical condition with life expectancy < 6 years
Sites / Locations
- Associated Retina ConsultantsRecruiting
- Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye InstituteRecruiting
- Ocugen Site 3 - Bascom Palmer Eye InstituteRecruiting
- Ocugen Site 6 - Emory UniversityRecruiting
- Ocugen Site 2 - Casey Eye Institute - OHSURecruiting
- Ocugen Site 8 - Mid Atlantic Retina - Wills Eye HospitalRecruiting
- Ocugen Site 1 - Retina Foundation of the SouthwestRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Experimental
Experimental
Experimental
Experimental
No Intervention
Experimental
Cohort 1 (Low Dose)
Cohort 2 (Mid Dose)
Cohort 3 (High Dose)
Pediatric Arm
Natural History Study (OCU400-104)
Adult Arm
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup and LCA patients with CEP290
Pediatric subjects will receive the medium dose concentration and will have subjects with RP and LCA
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)
Following DSMB confirmation, adult LCA subjects with CEP290 mutation will receive a medium dose concentration of OCU400.