A Phase 1/2 interventional study of OCU400 Low Dose and OCU400 Med Dose in Retinitis Pigmentosa and Leber Congenital Amaurosis, sponsored by Ocugen. Active, not recruiting at 7 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-07-31.
Sponsored by Ocugen · Phase 1/2, Interventional, and Treatment
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101). To document prospective eye pathology in the above subjects Investigators will also conduct a Natural History Study (OCU400-104)i
This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study.
This study will be conducted in two phases enrolling up to 24 subjects. Treated subjects will receive a single subretinal injection of OCU400 in the study eye.
This is a multicenter, open-label, dose-ranging study in two subgroups of subjects with three consecutive cohorts.
A total of 18 adult RP subjects from each of the following subgroups with Biallelic autosomal recessive NR2E3 mutations, autosomal dominant NR2E3 mutations or Autosomal dominant RHO mutations will be selected for dose escalation.
For the Phase I portion of the study, the 3+3 design for sequential dose-escalating cohorts will be used with scheduled 3 dosing levels between 9 and 18 subjects will be used to follow the design.
Up to 3 additional adult LCA patients with CEP290 mutations and at least 1 pediatric LCA subject, will be enrolled in the Phase 2 portion.
Sample Size Justification:
The trial will enroll up to 24 patients (18 adult RP, up to 3 LCA patients, and at least 1 pediatric LCA patient) in both Phase 1 and Phase 2 components.
Participants who meet eligibility criteria will be enrolled and receive a single subretinal injection of OCU400 in one study eye. Participants are considered to have completed this study if they complete the final EOS visit Week 48 (12 months following the IP dose). The study duration will be approximately 58 weeks for each participant and will be followed in Long Term Safety Follow Up for an additional 2 years.
Participants from the Phase 1/2 study who previously received the investigational product (OCU400) in one eye may be eligible to receive OCU400 in the untreated fellow eye, provided they meet the inclusion/exclusion criteria and have completed week 48 follow up visit.
Natural History Study (OCU400-104, 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 the earliest timepoint on or after the date of their diagnosis of RP or LCA. Enrollment for this study has closed.
268 studies on the registry are indexed under Retinitis Pigmentosa; 71 are open to participants now.
This study's enrollment of 22 is below the median of 27 across 173 interventional studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →Ocugen is the lead sponsor of 12 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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:
Exclusion Criteria for Adult RP:
Inclusion Criteria for Adult LCA:
Exclusion Criteria for Adult LCA:
Inclusion Criteria for Pediatric RP:
Exclusion Criteria for Pediatric RP:
Inclusion Criteria for Pediatric LCA:
Exclusion Criteria for Pediatric LCA:
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup
Drug: OCU400 Low Dose
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup
Drug: OCU400 Med Dose
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup
Drug: OCU400 High Dose
Pediatric subjects will receive the medium dose concentration
Drug: OCU400 Med Dose
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.
Drug: OCU400 Med Dose · Drug: OCU400 High Dose
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
Drug: OCU400 Med Dose · Drug: OCU400 High Dose
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.
Drug: OCU400 Second Eye Dosing
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)
subretinal injection of up to 1.66×10E10 vg/mL
subretinal injection of up to 3.33×10E10 vg/mL
subretinal injection of up to 1.66×10E11 vg/mL
subretinal injection of 1.0x10E11vg/mL in 250 μl
Study Drug-related adverse events (SDAE)
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.
Time frame: 1 year
Treatment-Emergent adverse events (TEAEs)
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.
Time frame: 1 year
Serious adverse events (SAEs)
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).
Time frame: 1 year
Best-corrected visual acuity (BCVA)
Measured as the ETDRS letter score on the EVA tester or E-ETDRS charts. Electronic ETDRS Visual Acuity Testing Protocol will be followed (confidential).
Time frame: 1 year (Changes from baseline)
Low-luminance visual acuity (LLVA)
Electronic Visual Acuity Tester (EVA) and a Sponsor specific Low-Luminance lens will be used. Early Treatment of Diabetic Retinopathy Study (ETDRS) will also be accepted as a backup.
Time frame: 1 year (Changes from baseline)
Slit-lamp biomicroscopy
Changes in visual function.
Time frame: 1 year (Changes from baseline)
Intraocular pressure (IOP)
IOP measurement by applanation or rebound tonometry. Confirmation with Goldmann tonometer if IOP reading is outside the normal range (8-21mmHg).
Time frame: 1 year (Changes from baseline)
Indirect ophthalmoscopy
If visual acuity is so poor that the participant is unable to count fingers or perceive hand motion, light perception will be tested with the indirect ophthalmoscope as the light source.
Time frame: 1 year (Changes from baseline)
anti-AAV5 (anti Adeno-associated virus type 5)
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.
Time frame: 1 year
anti-hNR2E3 antibodies (hNR2E3 gene)
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.
Time frame: 1 year
T-cell response
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.
Time frame: 1 year
Multi-luminance mobility testing (MLMT)
Subjects will navigate a standardized mobility maze under set conditions as specified times during the study. The mobility testing will follow a standardized administration and data acquisition protocol and may only be administered by site staff certified in the methodology.
Time frame: 1 year (Changes from baseline)
Changes in ellipsoid zone width/length on wide-field 20° SD-OCT
Ellipsoid zone area/outer segment length will be determined by Spectral Domain Optical Coherence Tomography (SD-OCT) using standardized systems and acquisition protocols.
Time frame: 1 year (Changes from baseline)
Contrast sensitivity
Contrast sensitivity will be conducted using Pelli-Robson chart.
Time frame: 1 year (Changes from baseline)
Full Field Light Stimulation Threshold (FST)
FST will be completed at scheduled times throughout the study period
Time frame: 1 year (Changes from baseline)
Photopic Static Visual Fields
The Octopus 900 will be used with a standardized white-on-white full field and a blue-on-yellow with full field
Time frame: 1 year (Changes from baseline)
Vision on Quality of Life
The National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25) and the Michigan Retinal Degeneration Questionnaire (MRDQ) questionnaires (for RP Adult subjects only) will be administered to assess the impact of vision on quality of subject's life.
Time frame: 1 year (Changes from baseline)
Full Field Electroretinogram
The International Society for Clinical Electrophysiology of Vision (ISCEV) guidelines will be followed for conducting ff-ERG (Full-field Electroretinography) for RP subjects only.
Time frame: 1 year (Changes from baseline)
Wide-field fundus autofluorescence (wf-FAF)
The intensity of FAF will be evaluated using 55° posterior pole scanning.
Time frame: 1 year (Changes from baseline)
Plan to share: No
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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