Status:
ACTIVE_NOT_RECRUITING
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Lead Sponsor:
Ocugen
Conditions:
Retinitis Pigmentosa
Leber Congenital Amaurosis
Eligibility:
All Genders
6+ years
Phase:
PHASE1
PHASE2
Brief 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 CEP29...
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...
Eligibility Criteria
Inclusion
- 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:
- 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.
- Exclusion Criteria for Adult RP:
- 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.
- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.
- Any medical condition with life expectancy \< 6 years.
- Inclusion Criteria for Adult LCA:
- Males or females at least 18 years of age at the time of informed consent.
- 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).
- Exclusion Criteria for Adult LCA:
- 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.
- Inclusion Criteria for Pediatric RP:
- 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.
- Exclusion Criteria for Pediatric RP:
- 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.
- Inclusion Criteria for Pediatric LCA:
- 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).
- Exclusion Criteria for Pediatric LCA:
- 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.
Exclusion
Key Trial Info
Start Date :
January 24 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2027
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT05203939
Start Date
January 24 2022
End Date
March 1 2027
Last Update
August 6 2025
Active Locations (7)
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1
Associated Retina Consultants
Phoenix, Arizona, United States, 85020
2
Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute
La Jolla, California, United States, 92093
3
Ocugen Site 3 - Bascom Palmer Eye Institute
Miami, Florida, United States, 33136
4
Ocugen Site 6 - Emory University
Atlanta, Georgia, United States, 30322