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
NCT ID: NCT06852963
Last Updated: 2025-12-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
16 participants
INTERVENTIONAL
2025-06-01
2028-03-03
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1: 30ug VP-001 every 8 weeks
VP-001
VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
Cohort 2: 75ug of VP-001 every 12 weeks
VP-001
VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
Interventions
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VP-001
VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
Eligibility Criteria
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Inclusion Criteria
* 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:
1. Mean microperimetry threshold: \>5 decibel (dB) to \<15 dB
2. Ellipsoid zone (EZ) length \>1000 microns of which 500 microns is contiguous, by SD-OCT
3. In the opinion of the Investigator, rod function is observed in any direction \>10 degrees per static perimetry at Screening Visit (Visit 1)
Exclusion Criteria
* 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.
18 Years
ALL
No
Sponsors
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PYC Therapeutics
INDUSTRY
Responsible Party
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Locations
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University of Florida College of Medicine
Jacksonville, Florida, United States
Bascom Palmer Eye Institute - University of Miami
Miami, Florida, United States
Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, United States
Casey Eye Institute - OHSU
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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VP001-CL103
Identifier Type: -
Identifier Source: org_study_id
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