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This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including participants previously treated with VP001 in the PLATYPUS Study or WALLABY Study for a minimum of 8 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: 30ug VP-001 every 8 weeks | Experimental |
| |
| Cohort 2: 75ug of VP-001 every 12 weeks | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VP-001 | Drug | VP-001 is an oligonucleotide-peptide conjugate administered intravitreally. |
|
| Measure | Description | Time Frame |
|---|---|---|
| To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy. | The incidence, severity, and relatedness of ocular TEAEs and TE-SAEs in the study eye over a 26-month time period for each of the repeat doses | 26 months |
| Measure | Description | Time Frame |
|---|---|---|
| To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts | To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. A gain of Early Treatment Diabetic Retinopathy Study (ETDRS) letters in Best-Corrected Visual Acuity (BCVA) from Baseline through EOS/ET would indicate vision improvement. |
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Key Inclusion Criteria:
Male or female sex; ≥18 years of age at Day 1/Baseline (Visit 2)
May have been previously enrolled in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol #VP001-CL102) study. At Screening Visit in this study, participants must have completed at least 8 weeks after last study agent administration in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol # VP001-CL102) study
Have a confirmed clinical diagnosis of Retinitis Pigmentosa.
Have a confirmed genetic diagnosis of Retinitis Pigmentosa secondary to mutation in the PRPF31 gene.
For participants not previously enrolled in VP001-CL101 or VP001-CL102 studies: Meet all of the following for visual function in the study eye at the Screening Visit:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida College of Medicine | Jacksonville | Florida | 32209 | United States | ||
| Bascom Palmer Eye Institute - University of Miami |
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| 24 months |
| To determine the change from baseline (Visit 2) through End of Study/Early Termination in Low Luminance Visual Acuity (LLVA) letter score | To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. | 24 months |
| Change from Baseline (Visit 2) through End of Study/Early Termination in visual field sensitivity, Mean deviation (Mean Defect) as measured by standard static perimetry (Humphries) | To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. | 24 months |
| Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity | To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. > mean retinal sensitivity will be measured by fundus-guided microperimetry, measured in dB (Decibels). | 24 months |
| Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT | SD-OCT refers to Spectral Domain Optical Coherence Tomography. A preserved EZ area refers to the region of the retina where the Ellipsoid Zone (EZ), a distinct hyperreflective band representing the inner segments of photoreceptor cells, remains intact and visible, indicating relatively healthy photoreceptor function, often measured in a specific area on the scan to monitor disease progression in conditions like retinitis pigmentosa. To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. | 24 months |
| Change from Baseline (Visit 2) through End of Study/Early Termination in participant reported outcome measures utilizing the Michigan Retinal Degeneration Questionnaire (MRDQ) | The Michigan Retinal Degeneration Questionnaire (MRDQ) is a psychometrically validated patient reported outcomes (PRO) measure for patients with inherited retinal degenerations. This is to determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. | 24 months |
| Miami |
| Florida |
| 33136 |
| United States |
| Kellogg Eye Center - University of Michigan | Ann Arbor | Michigan | 48105 | United States |
| Casey Eye Institute - OHSU | Portland | Oregon | 97239 | United States |
| Retina Foundation of the Southwest | Dallas | Texas | 75231 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| ID | Term |
|---|---|
| C563991 | Retinitis Pigmentosa 11 |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D015785 | Eye Diseases, Hereditary |
| D058499 | Retinal Dystrophies |
| ID | Term |
|---|---|
| D005128 | Eye Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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