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

Last updated: November 24, 2025
Sponsor: PYC Therapeutics
Overall Status: Active - Recruiting

Phase

1/2

Condition

Retinitis Pigmentosa

Eye Disorders/infections

Eye Disease

Treatment

VP-001

Clinical Study ID

NCT06852963
VP001-CL103
  • Ages > 18
  • All Genders

Study 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 retinal dystrophy, including participants previously treated with VP001 in the PLATYPUS Study or WALLABY Study for a minimum of 8 weeks.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • 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) orWALLABY (Protocol #VP001-CL102) study. At Screening Visit in this study,participants must have completed at least 8 weeks after last study agentadministration 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 inthe PRPF31 gene.

  • For participants not previously enrolled in VP001-CL101 or VP001-CL102 studies: Meetall 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, bySD-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

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 toinfection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemiccontrol issues, or any other medical condition that may put the participant at riskdue to study procedures.

  • Known mutations in genes that cause autosomal dominant RP, X-linked RP, or presenceof biallelic mutations in autosomal recessive RP/retinal dystrophy genes other thanPRPF31 mutations.

  • Have used anti-VEGF agents within 2 months or corticosteroid injections within thelast 3 months.

  • Have had Ozurdex® implants placed within 3 months or Retisert® or Iluvien® implantsplaced within 3 years prior to Baseline (Visit 2).

  • Within 3 months prior to Baseline (Visit 2), have undergone any vitreoretinalsurgery or any other ocular surgery

  • Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmicevaluation or photography, as assessed by the investigator.

  • Have used any investigational drug or device within 90 days or 5 estimatedhalf-lives (or within 60 days from last administration of VP-001 in the VP001-CL101Part B or VP001-CL102 studies) of Baseline (Visit 2), whichever is longer,

  • Have a recent history (<6 months) or current excessive recreational drug or alcoholuse, in the opinion of the investigator.

Study Design

Total Participants: 16
Treatment Group(s): 1
Primary Treatment: VP-001
Phase: 1/2
Study Start date:
June 01, 2025
Estimated Completion Date:
March 03, 2028

Connect with a study center

  • University of Florida College of Medicine

    Jacksonville, Florida 32209
    United States

    Site Not Available

  • Bascom Palmer Eye Institute - University of Miami

    Miami, Florida 33136
    United States

    Site Not Available

  • University of Florida College of Medicine

    Jacksonville 4160021, Florida 4155751 32209
    United States

    Active - Recruiting

  • Bascom Palmer Eye Institute - University of Miami

    Miami 4164138, Florida 4155751 33136
    United States

    Active - Recruiting

  • Kellogg Eye Center - University of Michigan

    Ann Arbor, Michigan 48105
    United States

    Site Not Available

  • Kellogg Eye Center - University of Michigan

    Ann Arbor 4984247, Michigan 5001836 48105
    United States

    Active - Recruiting

  • Casey Eye Institute - OHSU

    Portland, Oregon 97239
    United States

    Site Not Available

  • Casey Eye Institute - OHSU

    Portland 5746545, Oregon 5744337 97239
    United States

    Active - Recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas 75231
    United States

    Site Not Available

  • Baylor College of Medicine

    Houston, Texas 77030
    United States

    Site Not Available

  • Retina Foundation of the Southwest

    Dallas 4684888, Texas 4736286 75231
    United States

    Active - Recruiting

  • Baylor College of Medicine

    Houston 4699066, Texas 4736286 77030
    United States

    Active - Recruiting

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