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Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

Primary Purpose

Retinitis Pigmentosa, Leber Congenital Amaurosis

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
OCU400 Low Dose
OCU400 Med Dose
OCU400 High Dose
Sponsored by
Ocugen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring NR2E3, Rhodopsin, Enhanced S-cone syndrome, Cep290

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Arm Type

Experimental

Experimental

Experimental

Experimental

No Intervention

Experimental

Arm Label

Cohort 1 (Low Dose)

Cohort 2 (Mid Dose)

Cohort 3 (High Dose)

Pediatric Arm

Natural History Study (OCU400-104)

Adult Arm

Arm Description

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.

Outcomes

Primary Outcome Measures

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.
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.
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).

Secondary Outcome Measures

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).
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.
Slit-lamp biomicroscopy
Changes in visual function.
Intraocular pressure (IOP)
IOP measurement by applanation or rebound tonometry. Confirmation with Goldmann tonometer if IOP reading is outside the normal range (8-21mmHg).
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.
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.
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.
T-cell response
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.

Full Information

First Posted
November 16, 2021
Last Updated
October 24, 2023
Sponsor
Ocugen
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1. Study Identification

Unique Protocol Identification Number
NCT05203939
Brief Title
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 24, 2022 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ocugen

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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 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. 3 adult LCA patients with CEP290 mutations and 3 pediatric subjects with RP and LCA, will be enrolled in the Phase 2 portion. Sample Size Justification: The trial will enroll up to 24 patients (18 adult RP, 3 adult LCA, 2 pediatric RP, and 1 pediatric LCA) in both Phase 1 and Phase 2 components. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa, Leber Congenital Amaurosis
Keywords
NR2E3, Rhodopsin, Enhanced S-cone syndrome, Cep290

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
124 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1 (Low Dose)
Arm Type
Experimental
Arm Description
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup
Arm Title
Cohort 2 (Mid Dose)
Arm Type
Experimental
Arm Description
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup
Arm Title
Cohort 3 (High Dose)
Arm Type
Experimental
Arm Description
Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup and LCA patients with CEP290
Arm Title
Pediatric Arm
Arm Type
Experimental
Arm Description
Pediatric subjects will receive the medium dose concentration and will have subjects with RP and LCA
Arm Title
Natural History Study (OCU400-104)
Arm Type
No Intervention
Arm Description
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)
Arm Title
Adult Arm
Arm Type
Experimental
Arm Description
Following DSMB confirmation, adult LCA subjects with CEP290 mutation will receive a medium dose concentration of OCU400.
Intervention Type
Drug
Intervention Name(s)
OCU400 Low Dose
Intervention Description
subretinal injection of up to 1.66 × 10^10 vg/mL
Intervention Type
Drug
Intervention Name(s)
OCU400 Med Dose
Intervention Description
subretinal injection of upto 3.33 × 10^10 vg/mL
Intervention Type
Drug
Intervention Name(s)
OCU400 High Dose
Intervention Description
subretinal injection of upto 1.66 × 10^11 vg/mL
Primary Outcome Measure Information:
Title
Study Drug-related adverse events (SDAE)
Description
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
Title
Treatment-Emergent adverse events (TEAEs)
Description
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
Title
Serious adverse events (SAEs)
Description
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
Secondary Outcome Measure Information:
Title
Best-corrected visual acuity (BCVA)
Description
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)
Title
Low-luminance visual acuity (LLVA)
Description
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)
Title
Slit-lamp biomicroscopy
Description
Changes in visual function.
Time Frame
1 year (Changes from baseline)
Title
Intraocular pressure (IOP)
Description
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)
Title
Indirect ophthalmoscopy
Description
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)
Title
anti-AAV5 (anti Adeno-associated virus type 5)
Description
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.
Time Frame
1 year
Title
anti-hNR2E3 antibodies (hNR2E3 gene)
Description
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.
Time Frame
1 year
Title
T-cell response
Description
Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory.
Time Frame
1 year
Other Pre-specified Outcome Measures:
Title
Multi-luminance mobility testing (MLMT)
Description
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)
Title
Changes in ellipsoid zone width/length on wide-field 45° SD-OCT
Description
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)
Title
Contrast sensitivity
Description
Contrast sensitivity will be conducted using Pelli-Robson chart.
Time Frame
1 year (Changes from baseline)
Title
Full Field Light Stimulation Threshold (FST)
Description
FST will be completed at scheduled times throughout the study period
Time Frame
1 year (Changes from baseline)
Title
Static Visual Fields
Description
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)
Title
Vision on Quality of Life
Description
The National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25) and the Michigan Retinal Degeneration Questionnaire (MRDQ) questionnaires will be administered to assess the impact of vision on quality of subject's life.
Time Frame
1 year (Changes from baseline)
Title
Full Field Electroretinogram
Description
The International Society for Clinical Electrophysiology of Vision (ISCEV) guidelines will be followed for conducting ff-ERG (Full-field Electroretinography)
Time Frame
1 year (Changes from baseline)
Title
Wide-field fundus autofluorescence (wf-FAF)
Description
The intensity of FAF will be evaluated using 55° posterior pole scanning.
Time Frame
1 year (Changes from baseline)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
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 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 subjects in Cohort 1-3, BCVA ≤ 20/50 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye. Unable to perform a Multi-Luminance Mobility Testing (MLMT) using study eye 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. 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 3.5 (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 Any contraindications for subretinal injection. Any intraocular surgery within 6 months. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis) 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). 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. 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) 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 3.5 (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 Any contraindications for subretinal injection. 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Murthy Chavali, PhD
Phone
405-714-4198
Email
murthy.chavali@ocugen.com
First Name & Middle Initial & Last Name or Official Title & Degree
Sahar Matloob, MD, ACRP-CP
Phone
610-871-6270
Email
sahar.matloob@ocugen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Huma Qamar, MD, MPH, CMI
Organizational Affiliation
Ocugen
Official's Role
Study Director
Facility Information:
Facility Name
Associated Retina Consultants
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85020
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Benjamin Bakall
Facility Name
Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92093
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Borooah
Facility Name
Ocugen Site 3 - Bascom Palmer Eye Institute
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lam
Facility Name
Ocugen Site 6 - Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jain
Facility Name
Ocugen Site 2 - Casey Eye Institute - OHSU
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yang
Facility Name
Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pulido
Facility Name
Ocugen Site 1 - Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kirsten Locke
First Name & Middle Initial & Last Name & Degree
David Birch, PhD

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
32123325
Citation
Li S, Datta S, Brabbit E, Love Z, Woytowicz V, Flattery K, Capri J, Yao K, Wu S, Imboden M, Upadhyay A, Arumugham R, Thoreson WB, DeAngelis MM, Haider NB. Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa. Gene Ther. 2021 May;28(5):223-241. doi: 10.1038/s41434-020-0134-z. Epub 2020 Mar 2.
Results Reference
background
Links:
URL
https://pubmed.ncbi.nlm.nih.gov/32123325/
Description
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa

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Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

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