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NCT06852963

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

Active, Not Recruiting
PHASE1Ages 18+InterventionalTreatment
PYC Therapeutics
~17 participants
Updated 2026-05-29 on ClinicalTrials.gov
What's tested:VP-001

At a glance

Recruiting sites
0 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy.
Measured over 26 months

Conditions

Retinitis Pigmentosa 11
Retinal Degeneration
Retinal Disease
Eye Diseases Hereditary
Retinal Dystrophies

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NCT06852963

Where you'd take part

This study runs at 6 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Bascom Palmer Eye Institute - University of Miami

    Miami, Floridano site contact published

  • Baylor College of Medicine

    Houston, Texasno site contact published

  • Casey Eye Institute - OHSU

    Portland, Oregonno site contact published

  • Kellogg Eye Center - University of Michigan

    Ann Arbor, Michiganno site contact published

  • Retina Foundation of the Southwest

    Dallas, Texasno site contact published

  • University of Florida College of Medicine

    Jacksonville, Floridano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

Male or female sex; ≥18 years of age at Day 1/Baseline (Visit 2)
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:

Exclusion

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.

What this trial measures

  • To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy.26 months

    The incidence, severity, and relatedness of ocular TEAEs and TE-SAEs in the study eye over a 26-month time period for each of the repeat doses