A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 (30 μg and 75 μg) Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 in the PLATYPUS Study (Protocol # VP001-101) or WALLABY Study (Protocol # VP001-102) for a Minimum of 8 Weeks
PYC Therapeutics
Summary
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.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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 particip…
Interventions
- DrugVP-001
VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
Locations (6)
- University of Florida College of MedicineJacksonville, Florida
- Bascom Palmer Eye Institute - University of MiamiMiami, Florida
- Kellogg Eye Center - University of MichiganAnn Arbor, Michigan
- Casey Eye Institute - OHSUPortland, Oregon
- Retina Foundation of the SouthwestDallas, Texas
- Baylor College of MedicineHouston, Texas