Trials / Recruiting
RecruitingNCT06852963
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
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
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 16 (estimated)
- Sponsor
- PYC Therapeutics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
- Retinitis Pigmentosa 11
- Retinal Degeneration
- Retinal Disease
- Eye Diseases Hereditary
- Retinal Dystrophies
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | VP-001 | VP-001 is an oligonucleotide-peptide conjugate administered intravitreally. |
Timeline
- Start date
- 2025-06-01
- Primary completion
- 2028-01-30
- Completion
- 2028-03-03
- First posted
- 2025-02-28
- Last updated
- 2025-12-02
Locations
6 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06852963. Inclusion in this directory is not an endorsement.