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 (DINGO)

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 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.

Study Overview

• Status: Active, not recruiting
• Conditions: Retinal Degeneration; Retinal Dystrophies; Retinal Disease; Retinitis Pigmentosa 11; Eye Diseases Hereditary
• Intervention / Treatment: Drug: VP-001

• Study Type: Interventional
• Enrollment (Actual): 17
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Jacksonville, Florida, United States, 32209
      • University of Florida College of Medicine
    • Miami, Florida, United States, 33136
      • Bascom Palmer Eye Institute - University of Miami
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Kellogg Eye Center - University of Michigan
  • Oregon
    • Portland, Oregon, United States, 97239
      • Casey Eye Institute - OHSU
  • Texas
    • Dallas, Texas, United States, 75231
      • Retina Foundation of the Southwest
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: 30ug VP-001 every 8 weeks	Drug: VP-001; VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
Experimental: Cohort 2: 75ug of VP-001 every 12 weeks	Drug: VP-001; VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts	To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. A gain of Early Treatment Diabetic Retinopathy Study (ETDRS) letters in Best-Corrected Visual Acuity (BCVA) from Baseline through EOS/ET would indicate vision improvement.	24 months
To determine the change from baseline (Visit 2) through End of Study/Early Termination in Low Luminance Visual Acuity (LLVA) letter score	To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.	24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in visual field sensitivity, Mean deviation (Mean Defect) as measured by standard static perimetry (Humphries)	To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.	24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity	To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. > mean retinal sensitivity will be measured by fundus-guided microperimetry, measured in dB (Decibels).	24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT	SD-OCT refers to Spectral Domain Optical Coherence Tomography. A preserved EZ area refers to the region of the retina where the Ellipsoid Zone (EZ), a distinct hyperreflective band representing the inner segments of photoreceptor cells, remains intact and visible, indicating relatively healthy photoreceptor function, often measured in a specific area on the scan to monitor disease progression in conditions like retinitis pigmentosa. To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.	24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in participant reported outcome measures utilizing the Michigan Retinal Degeneration Questionnaire (MRDQ)	The Michigan Retinal Degeneration Questionnaire (MRDQ) is a psychometrically validated patient reported outcomes (PRO) measure for patients with inherited retinal degenerations. This is to determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.	24 months

Collaborators and Investigators

• Sponsor: PYC Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2025
• Primary Completion (Estimated): March 8, 2028
• Study Completion (Estimated): April 21, 2028

Study Registration Dates

• First Submitted: February 20, 2025
• First Submitted That Met QC Criteria: February 24, 2025
• First Posted (Actual): February 28, 2025

Study Record Updates

• Last Update Posted (Actual): May 29, 2026
• Last Update Submitted That Met QC Criteria: May 27, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: VP-001-CL103
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinal Diseases; Retinal Degeneration; Retinal Dystrophies; Eye Diseases, Hereditary; Retinitis Pigmentosa 11
• Other Study ID Numbers: VP001-CL103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No