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RECRUITING
NCT06852963
PHASE1/PHASE2

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

View on ClinicalTrials.gov

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

Interventions

DRUG

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