Status:
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
Lead Sponsor:
PYC Therapeutics
Conditions:
Retinitis Pigmentosa 11
Retinal Degeneration
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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 retina...
Eligibility Criteria
Key Inclusion Criteria:
-
Male or female sex; ≥18 years of age at Day 1/Baseline (Visit 2)
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May have been previously enrolled in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol #VP001-CL102) study. At Screening Visit in this study, participants must have completed at least 8 weeks after last study agent administration in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol # VP001-CL102) study
-
Have a confirmed clinical diagnosis of Retinitis Pigmentosa.
-
Have a confirmed genetic diagnosis of Retinitis Pigmentosa secondary to mutation in the PRPF31 gene.
-
For participants not previously enrolled in VP001-CL101 or VP001-CL102 studies: Meet all of the following for visual function in the study eye at the Screening Visit:
- Mean microperimetry threshold: >5 decibel (dB) to <15 dB
- Ellipsoid zone (EZ) length >1000 microns of which 500 microns is contiguous, by SD-OCT
- In the opinion of the Investigator, rod function is observed in any direction >10 degrees per static perimetry at Screening Visit (Visit 1)
Key Exclusion Criteria:
- Have any uncontrolled systemic disease that, in the opinion of the Investigator, would preclude participation in the study that include but are not limited to infection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemic control issues, or any other medical condition that may put the participant at risk due to study procedures.
- Known mutations in genes that cause autosomal dominant RP, X-linked RP, or presence of biallelic mutations in autosomal recessive RP/retinal dystrophy genes other than PRPF31 mutations.
- Have used anti-VEGF agents within 2 months or corticosteroid injections within the last 3 months.
- Have had Ozurdex® implants placed within 3 months or Retisert® or Iluvien® implants placed within 3 years prior to Baseline (Visit 2).
- Within 3 months prior to Baseline (Visit 2), have undergone any vitreoretinal surgery or any other ocular surgery
- Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the investigator.
- Have used any investigational drug or device within 90 days or 5 estimated half-lives (or within 60 days from last administration of VP-001 in the VP001-CL101 Part B or VP001-CL102 studies) of Baseline (Visit 2), whichever is longer,
- Have a recent history (<6 months) or current excessive recreational drug or alcohol use, in the opinion of the investigator.
Key Trial Info
Start Date :
June 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 3 2028
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT06852963
Start Date
June 1 2025
End Date
March 3 2028
Last Update
December 2 2025
Active Locations (6)
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1
University of Florida College of Medicine
Jacksonville, Florida, United States, 32209
2
Bascom Palmer Eye Institute - University of Miami
Miami, Florida, United States, 33136
3
Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, United States, 48105
4
Casey Eye Institute - OHSU
Portland, Oregon, United States, 97239