drugset / Trial / 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
Non-randomizedParallel-groupOpen-labelTreatment
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.
Timeline
- Start
- 2025-06-01
- Primary completion
- 2028-03-08
- Completion
- 2028-04-21
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VP-001 | Oligonucleotide (other) | 30 ug | Intravitreal |
| Subject | VP-001 | Oligonucleotide (other) | 75 ug | Intravitreal |