Clinical Research Directory
Browse clinical research sites, groups, and studies.
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
Sponsor: 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.
Official title: 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
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
16
Start Date
2025-06-01
Completion Date
2028-03-03
Last Updated
2025-12-02
Healthy Volunteers
No
Conditions
Interventions
VP-001
VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
Locations (6)
University of Florida College of Medicine
Jacksonville, Florida, United States
Bascom Palmer Eye Institute - University of Miami
Miami, Florida, United States
Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, United States
Casey Eye Institute - OHSU
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Baylor College of Medicine
Houston, Texas, United States