Trials / NCT06852963
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001
Underway, not enrolling Phase 1 / Phase 2 RNA / antisense
- Sponsor
- PYC Therapeutics
- Treatment
- VP-001
- Gene
- PRPF31
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Retinal Disease, Eye Diseases Hereditary, Retinal Dystrophies
- Ages
- 18 Years to no upper limit
- Enrollment
- 17 participants
- Started
- 2025-06-01
- Main results expected
- 2028-03-08
- Results posted
- Not yet
- Record last updated
- 2026-05-29
What the study is testing
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.
Where it runs
United States (6)
- University of Florida College of Medicine, Jacksonville, Florida
- Bascom Palmer Eye Institute - University of Miami, Miami, Florida
- Kellogg Eye Center - University of Michigan, Ann Arbor, Michigan
- Casey Eye Institute - OHSU, Portland, Oregon
- Retina Foundation of the Southwest, Dallas, Texas
- Baylor College of Medicine, Houston, Texas