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This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP.
This is a multicenter, assessor blinded and randomized study which will enroll 140 subjects. Study has completed enrollment of all 140 subjects.
A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.
Subjects in each arm will be randomized into treatment and control groups with a 2:1 ratio. Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RHO Arm | Experimental | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye |
|
| Gene Agnostic Arm | Experimental | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye |
|
| Control for RHO Arm | No Intervention | Will not receive any active study intervention | |
| Control for Gene Agnostic Arm | No Intervention | Will not receive any active study intervention |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sub-Retinal Administration of OCU400-301 | Genetic | Sub-Retinal Administration of OCU400-301 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control | Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA) | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group | Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA). |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in Patients Global Impression of Change (PGIC) score | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Change from baseline in Patients Global Impression of Change (PGIC) score. | 52 weeks |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Huma Qamar | Ocugen | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Associated Retina Consultants | Phoenix | Arizona | 85020 | United States | ||
| University of Southern Califormia |
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This is outcome assessor blinded study
| 52 weeks |
| Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls | Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls in all RP subjects, as assessed by binocular low luminance visual acuity when using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score at Week 52. | 52 weeks |
| Change from baseline in LDNA Lux-level results in treated subjects when compared to the untreated controls | Change from baseline in LDNA Lux-level results in all RP subjects in treated subjects when compared to the untreated controls at weeks 12, 24, 36, and 52 | 52 weeks |
| Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores | Change in LLVA letter scores in Gene Agnostic Arm subjects will be compared to controls | 52 weeks |
| Change from baseline in Best corrected visual acuity (BCVA) measured by ETDRS | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Change from baseline in Best corrected visual acuity (BCVA) measured by ETDRS. | 52 weeks |
| Improvement in Low luminance deficit (LLD) from baseline measured by ETDRS | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Improvement in Low luminance deficit (LLD) from baseline measured by ETDRS. | 52 weeks |
| Improvement in mean retinal sensitivity from baseline as measured by static perimetry in the 30-degree | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Improvement in mean retinal sensitivity from baseline as measured by static perimetry in the 30-degree. | 52 weeks |
| Change from baseline in hyperfluorescent ring as measured by Wide field-FAF | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Change from baseline in hyperfluorescent ring as measured by Wide field-FAF. | 52 weeks |
| Change from baseline in area of RP atrophy in macular region as measured by Wide field-FAF | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Change from baseline in area of RP atrophy in macular region as measured by Wide field-FAF. | 52 weeks |
| Change from baseline in hypo autofluorescence assessments in peripheral retina as measured by Wide field-FAF | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by: Change from baseline in hypo autofluorescence assessments in peripheral retina as measured by Wide field-FAF. | 52 weeks |
| Change from baseline in hyper autofluorescence assessments in peripheral retina as measured by Wide field-FAF | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by change from baseline in hyper autofluorescence assessments in peripheral retina as measured by Wide field-FAF. | 52 weeks |
| Changes in the Ellipsoid zone area from baseline as measured by SD-OCT | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by changes in Ellipsoid zone area from baseline as measured by SD-OCT. | 52 weeks |
| Changes in the outer segment length from baseline as measured by SD-OCT | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by changes in the outer segment length as measured by SD-OCT. | 52 weeks |
| Changes in the outer segment volume from baseline as measured by SD-OCT | Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by changes in the outer segment volume from baseline as measured by SD-OCT. | 52 weeks |
| La Jolla |
| California |
| 92093 |
| United States |
| University of Southern California, Roski Eye Insitute | Los Angeles | California | 90033 | United States |
| Advanced Research, LLC. | Deerfield Beach | Florida | 33064 | United States |
| Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine | Miami | Florida | 33136 | United States |
| Erie Retina Research LLC | Erie | Pennsylvania | 16507 | United States |
| Vanderbilt Eye Institute | Nashville | Tennessee | 37232 | United States |
| Retina Consultants of Texas | Bellaire | Texas | 77401 | United States |
| Retina Foundation of the Southwest | Dallas | Texas | 75231 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Valley Retina Institute | McAllen | Texas | 78503 | United States |
| Gundersen Health System | La Crosse | Wisconsin | 54601 | United States |
| Calgary Retina Consultants | Calgary | Alberta | T2H OC8 | Canada |
| University of Alberta | Edmonton | Alberta | T6G 3E1 | Canada |
| The University of British Columbia | Vancouver | British Columbia | V5Z 3N9 | Canada |
| Sunnybrook Health Sciences Centre | Toronto | Ontario | M4N 3M5 | Canada |
| Research Institute of the McGill University Health Centre | Montreal | Quebec | H2W 1L4 | Canada |
| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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