Retinitis PigmentosaPhase 1/2NCT05203939
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
A Phase 1/2 study testing OCU400 Low Dose, OCU400 Med Dose and OCU400 High Dose in people with Retinitis Pigmentosa and Leber Congenital Amaurosis, run by Ocugen at 7 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Leber Congenital Amaurosis
- What is being tested?
- OCU400 Low Dose, OCU400 Med Dose and OCU400 High Dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 7 sitesUnited States
- Study dates
- 24 Jan 2022to Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males or females ≥ 18 years of age at the time of informed consent.
- Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
- 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.
- Males or females at least 18 years of age at the time of informed consent.
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- Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
- Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.8 (light perception) in the study eye.
- Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).
- Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
- Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
- BCVA ≤ 20/32 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.
- Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
- Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
- Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.8 (light perception) in the study eye.
- Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).
Who cannot join
- Subject lacks evidence of outer nuclear layer.
- 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.
- 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.
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- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.
- Any medical condition with life expectancy < 6 years.
- Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function.
- Considered unsuitable for any reason that may either place the patient at increased risk during participation or interfere with the interpretation of the study safety and efficacy outcomes by the Investigator, after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
- Any contraindications for subretinal injection.
- Any intraocular surgery within 6 months.
- Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.
- Subject lacks evidence of outer nuclear layer 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.
- 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, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.
- Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
- Any medical condition with life expectancy < 6 years.
- Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function.
- Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study safety and efficacy outcomes by the Investigator, after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
- Any contraindications for subretinal injection.
- Any Intraocular surgery within 6 months.
- Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
- Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Ocugen Site 6 - Emory University
Atlanta, Georgia, 30322
Ocugen Site 1 - Retina Foundation of the Southwest
Dallas, Texas, 75231
Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute
La Jolla, California, 92093
Ocugen Site 3 - Bascom Palmer Eye Institute
Miami, Florida, 33136
Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital
Philadelphia, Pennsylvania, 19107
Associated Retina Consultants
Phoenix, Arizona, 85020
Ocugen Site 2 - Casey Eye Institute - OHSU
Portland, Oregon, 97239
About this study
Official title: A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 and RHO Mutations and Leber Congenital Amaurosis With Mutation(s) in CEP290 Gene
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.
Read the detailed description
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.
- Conditions
- Retinitis Pigmentosa, Leber Congenital Amaurosis
- Being tested
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing
- Keywords
- NR2E3, Rhodopsin, Enhanced S-cone syndrome, Cep290
- Medical subject headings
- Retinitis Pigmentosa, Leber Congenital Amaurosis, Night Blindness, Congenital Stationary, Autosomal Dominant 1, Enhanced S-Cone Syndrome, Meckel Syndrome, Type 4
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OCU400 Low DoseDrug
subretinal injection of up to 1.66×10E10 vg/mL
OCU400 Med DoseDrug
subretinal injection of up to 3.33×10E10 vg/mL
OCU400 High DoseDrug
subretinal injection of up to 1.66×10E11 vg/mL
OCU400 Second Eye DosingDrug
subretinal injection of 1.0x10E11vg/mL in 250 μl
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Low Dose) | Experimental | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup Receives: Drug: OCU400 Low Dose |
| Cohort 2 (Mid Dose) | Experimental | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup Receives: Drug: OCU400 Med Dose |
| Cohort 3 (High Dose) | Experimental | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup Receives: Drug: OCU400 High Dose |
| Pediatric Arm | Experimental | Pediatric subjects will receive the medium dose concentration Receives: Drug: OCU400 Med Dose |
| 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. Receives: Drug: OCU400 Med Dose; Drug: OCU400 High Dose |
| 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 Receives: Drug: OCU400 Med Dose; Drug: OCU400 High Dose |
| 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. Receives: Drug: OCU400 Second Eye Dosing |
| 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) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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).
Measured: 1 year
Secondary outcomes (8)
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).
Measured: 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.
Measured: 1 year (Changes from baseline)
Slit-lamp biomicroscopy
Changes in visual function.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2022
- Study start24 Jan 2022
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ocugen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jennifer Henrick, RN, MS, Study director, OcugenMohamed Genead, MD, Study chair, Ocugen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05203939
- Sponsor's protocol ID
- OCU400-101
- Status last verified
- Jul 2026
- First submitted
- 16 Nov 2021
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 and RHO Mutations and Leber Congenital Amaurosis With Mutation(s) in CEP290 Gene
- ClinicalTrials.gov ID
- NCT05203939
- Acronym / sponsor ID
- OCU400-101
- Conditions
- Retinitis Pigmentosa, Leber Congenital Amaurosis
- Keywords
- NR2E3, Rhodopsin, Enhanced S-cone syndrome, Cep290
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 22 participants (actual)
- Lead sponsor
- OcugenIndustry (pharma, biotech, devices)
- Study start
- 24 Jan 2022 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 24 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Low Dose) | Experimental | Drug: OCU400 Low DoseBiallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup |
| Cohort 2 (Mid Dose) | Experimental | Drug: OCU400 Med DoseBiallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup |
| Cohort 3 (High Dose) | Experimental | Drug: OCU400 High DoseBiallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup |
| Pediatric Arm | Experimental | Drug: OCU400 Med DosePediatric subjects will receive the medium dose concentration |
| Phase 2 (High and Medium Dose) | Experimental | Drug: OCU400 Med Dose, Drug: OCU400 High DoseFollowing 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 | Drug: OCU400 Med Dose, Drug: OCU400 High DoseBiallelic 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 | Drug: OCU400 Second Eye DosingEligible 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) |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- Males or females ≥ 18 years of age at the time of informed consent.
- Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
- 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.
Key exclusion criteria (29 listed)
- Subject lacks evidence of outer nuclear layer.
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OCU400 Low Dosesubretinal injection of up to 1.66×10E10 vg/mL | Drug | Cohort 1 (Low Dose) |
| OCU400 Med Dosesubretinal injection of up to 3.33×10E10 vg/mL | Drug | Adult Arm, Cohort 2 (Mid Dose), Pediatric Arm, Phase 2 (High and Medium Dose) |
| OCU400 High Dosesubretinal injection of up to 1.66×10E11 vg/mL | Drug | Adult Arm, Cohort 3 (High Dose), Phase 2 (High and Medium Dose) |
| OCU400 Second Eye Dosingsubretinal injection of 1.0x10E11vg/mL in 250 μl | Drug | Second Eye Dosing |
Primary outcome
Study Drug-related adverse events (SDAE)
Time frame: 1 year
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.
Treatment-Emergent adverse events (TEAEs)
Time frame: 1 year
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.
Serious adverse events (SAEs)
Time frame: 1 year
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).
8 secondary outcomes
Best-corrected visual acuity (BCVA)
Time frame: 1 year (Changes from baseline)
Low-luminance visual acuity (LLVA)
Time frame: 1 year (Changes from baseline)
Slit-lamp biomicroscopy
Time frame: 1 year (Changes from baseline)
Intraocular pressure (IOP)
Time frame: 1 year (Changes from baseline)
Indirect ophthalmoscopy
Time frame: 1 year (Changes from baseline)
anti-AAV5 (anti Adeno-associated virus type 5)
Time frame: 1 year
anti-hNR2E3 antibodies (hNR2E3 gene)
Time frame: 1 year
T-cell response
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Retina World Congress 2024- Meeting Abstract. Title: Safety and Efficacy Results from a Phase 1/2 Clinical Trial of OCU400 Modifier Gene Therapy for Treatment of Retinitis PigmentosaLink provided by the sponsor.
- International Conference on Ophthalmology and Vision Science, Canada- Meeting Abstract: Title: Evaluation of Safety and Efficacy of OCU400 Gene Therapy for Retinitis Pigmentosa: Phase 1/2 Study ResultsLink provided by the sponsor.
- American Academy of Ophthalmology (AAO) Annual Meeting- Title: Safety and Efficacy of OCU400 Gene Modifier Therapy for Retinitis Pigmentosa: Phase 1/2 Study UpdatesLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jennifer Henrick, RN, MS study director
Ocugen
Mohamed Genead, MD study chair
Ocugen
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing OCU400 Low Dose, OCU400 Med Dose and OCU400 High Dose in people with Retinitis Pigmentosa and Leber Congenital Amaurosis, run by Ocugen at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Study Drug-related adverse events (SDAE) (time frame: 1 year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2026.