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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Retinitis Pigmentosa 11, Retinal Degeneration, Retinal Disease, Eye Diseases Hereditary, Retinal Dystrophies

In brief

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.

Key facts

Study ID

NCT06852963

Run by

PYC Therapeutics

People needed

17

Starts

2025-06-01

Expected to finish

2028-04-21

Last updated by the study team

2026-05-29

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• 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

Full record on ClinicalTrials.gov

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