Actively Recruiting
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
Led by PYC Therapeutics · Updated on 2025-12-02
16
Participants Needed
6
Research Sites
143 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female, 18 years or older at Day 1/Baseline
- May have previously participated in the PLATYPUS Part B or WALLABY study, with at least 8 weeks since last VP-001 dose
- Confirmed clinical diagnosis of Retinitis Pigmentosa
- Confirmed genetic diagnosis of Retinitis Pigmentosa due to PRPF31 mutation
- For new participants: Visual function in study eye with mean microperimetry threshold >5 dB and <15 dB
- Ellipsoid zone length >1000 microns with at least 500 microns contiguous by SD-OCT
- Investigator observes rod function >10 degrees per static perimetry at Screening
You will not qualify if you...
- Any uncontrolled systemic disease that could increase risk from study procedures, including infection, uncontrolled high blood pressure, cardiovascular disease, or poor glycemic control
- Known mutations causing autosomal dominant or X-linked Retinitis Pigmentosa, or biallelic mutations in autosomal recessive RP genes other than PRPF31
- Use of anti-VEGF agents within 2 months or corticosteroid injections within 3 months
- Ozurdex implants within 3 months, or Retisert or Iluvien implants within 3 years before Baseline
- Vitreoretinal or other ocular surgery within 3 months before Baseline
- Ocular media opacity or poor pupil dilation preventing quality eye exams or photography
- Use of any investigational drug or device within 90 days or 5 half-lives (or within 60 days if from previous VP-001 studies) before Baseline
- Recent or current excessive recreational drug or alcohol use within 6 months
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
University of Florida College of Medicine
Jacksonville, Florida, United States, 32209
Actively Recruiting
2
Bascom Palmer Eye Institute - University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
3
Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, United States, 48105
Actively Recruiting
4
Casey Eye Institute - OHSU
Portland, Oregon, United States, 97239
Actively Recruiting
5
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
6
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
O
Ora Inc
CONTACT
J
Jessica Dunne
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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