Actively Recruiting
Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With PRPF31 Mutation-Associated Retinal Dystrophy Including Those Previously Treated With VP-001
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
Researchers are evaluating the safety and effectiveness of two doses of VP-001, an oligonucleotide-peptide conjugate given by injection into the eye, for people with retinal dystrophy caused by mutations in the PRPF31 gene. This Phase 1/2 open-label study includes participants who have been previously treated with VP-001 in earlier studies, as well as new participants, to better understand treatment effects and safety over time. The study involves two groups receiving different doses of VP-001 (30 micrograms and 75 micrograms) administered intravitreally. Participants may have been part of previous VP-001 studies and must have waited at least 8 weeks since their last dose before joining this trial. Researchers will monitor these participants over 26 months to assess repeated dosing safety. During the study, participants will undergo various eye exams and imaging tests to evaluate visual function and retinal health. Safety will be closely observed through clinical assessments and monitoring for any side effects during the 26-month follow-up period. The main goal is to measure the safety of repeated VP-001 doses in people with confirmed PRPF31 mutation-associated retinal dystrophy.
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
J
Jessica Dunne
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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