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

NCT06852963 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: VP001-CL103
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 6

Brief 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.

Conditions

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

Keywords

VP-001-CL103

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts

  24 months

• To determine the change from baseline (Visit 2) through End of Study/Early Termination in Low Luminance Visual Acuity (LLVA) letter score

  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)

  24 months

• Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity

  24 months

• Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT

  24 months

Study Arms (2)

Cohort 1: 30ug VP-001 every 8 weeks

EXPERIMENTAL

Drug: VP-001

Cohort 2: 75ug of VP-001 every 12 weeks

EXPERIMENTAL

Drug: VP-001

Interventions

VP-001 DRUG

VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.

Cohort 1: 30ug VP-001 every 8 weeks; Cohort 2: 75ug of VP-001 every 12 weeks

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Mean microperimetry threshold: \>5 decibel (dB) to \<15 dB
• Ellipsoid zone (EZ) length \>1000 microns of which 500 microns is contiguous, by SD-OCT
• In the opinion of the Investigator, rod function is observed in any direction \>10 degrees per static perimetry at Screening Visit (Visit 1)

You may not qualify if:

• 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.

Sponsors & Collaborators

• PYC Therapeutics: lead

Study Sites (6)

University of Florida College of Medicine

Jacksonville, Florida, 32209, United States

Location

Bascom Palmer Eye Institute - University of Miami

Miami, Florida, 33136, United States

Location

Kellogg Eye Center - University of Michigan

Ann Arbor, Michigan, 48105, United States

Location

Casey Eye Institute - OHSU

Portland, Oregon, 97239, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa 11; Retinal Degeneration; Retinal Diseases; Eye Diseases, Hereditary; Retinal Dystrophies

Condition Hierarchy (Ancestors)

Eye Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2025
• First Posted: February 28, 2025
• Study Start: June 1, 2025
• Primary Completion (Estimated): March 8, 2028
• Study Completion (Estimated): April 21, 2028
• Last Updated: May 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)