Repeat-dose intravitreal VP-001 for PRPF31-related retinal dystrophy (two dose levels)

A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 (30 μg and 75 μg) Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 in the PLATYPUS Study (Protocol # VP001-101) or WALLABY Study (Protocol # VP001-102) for a Minimum of 8 Weeks

This trial will test two doses of VP-001 injected into the eye to see if it is safe and helps adults with PRPF31 mutation–related retinitis pigmentosa, including people who were treated with VP‑001 before.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 16 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: PYC Therapeutics (industry)
• Locations: 6 sites (Jacksonville, Florida and 5 other locations)
• Trial ID: NCT06852963 on ClinicalTrials.gov

What this trial studies

This is an open-label, parallel two-arm Phase 1/2 study testing repeat intravitreal doses of VP-001 at 30 µg and 75 µg in adults with genetically confirmed PRPF31-associated retinal dystrophy. Participants may include people previously treated with VP‑001 in earlier studies if at least eight weeks have passed since their last dose. The trial will follow safety and efficacy outcomes over time using visual function tests such as microperimetry, structural OCT measures including ellipsoid zone length, and other standard ophthalmic assessments. The design compares two dose levels to characterize tolerability and potential functional benefit and to inform further development.

Who should consider this trial

Good fit: Adults (≥18) with a confirmed diagnosis of retinitis pigmentosa caused by a PRPF31 mutation, including those previously treated with VP‑001 who meet the timing requirement, and new participants with measurable retinal structure and function per the study criteria.

Not a fit: People without a PRPF31 mutation or with retinal disease too advanced to meet the study's structural or functional entry criteria are unlikely to benefit from this treatment.

Why it matters

Potential benefit: If successful, the treatment could slow vision loss or improve retinal function in people with PRPF31 mutation–related retinitis pigmentosa.

How similar studies have performed: Some gene‑targeted treatments for inherited retinal diseases have shown benefits in other genetic subtypes, but repeat‑dose intravitreal therapy specifically targeting PRPF31 is relatively novel and early in clinical testing.

Eligibility criteria

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) 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)

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.

Where this trial is running

Jacksonville, Florida and 5 other locations

• University of Florida College of Medicine — Jacksonville, Florida, United States (RECRUITING)
• Bascom Palmer Eye Institute - University of Miami — Miami, Florida, United States (RECRUITING)
• Kellogg Eye Center - University of Michigan — Ann Arbor, Michigan, United States (RECRUITING)
• Casey Eye Institute - OHSU — Portland, Oregon, United States (RECRUITING)
• Retina Foundation of the Southwest — Dallas, Texas, United States (RECRUITING)
• Baylor College of Medicine — Houston, Texas, United States (RECRUITING)

Study contacts

• Study coordinator: Ora Inc

Conditions: Retinitis Pigmentosa 11, Retinal Degeneration, Retinal Disease, Eye Diseases Hereditary, Retinal Dystrophies, VP-001-CL103