SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects

NCT05902962 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: VP001-CL101
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 6

Brief Summary

A Phase 1 Open-Label, Single Arm Dose Escalation Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

Conditions

Retinal Dystrophy; PRPF31 Mutationassociated Retinal Dystrophy; RP11

Outcome Measures

Primary Outcomes (2)

• The incidence, severity, and relatedness of treatment-emergent ocular adverse events (TEAEs) and treatment-emergent serious adverse events (TE-SAEs) in the study eye

  over a 24-week time period

• The incidence, severity, and relatedness of treatment-emergent ocular adverse events (TEAEs) and treatment-emergent serious adverse events (TE-SAEs) in the study eye

  over a 48-week time period

Secondary Outcomes (4)

• Adverse Events and Treatment Emergent Serious adverse events (SAEs) in the fellow eye

  over a 24-week time period

• Adverse Events and Treatment Emergent serious adverse events (SAEs) in the fellow eye

  over a 48-week time period

• Incidence, severity relatedness and of non-ocular TEAEs

  over a 24-week time period

• Incidence, severity relatedness and of non-ocular TEAEs

  over a 48-week time period

Other Outcomes (24)

• Change from Baseline in Best-corrected visual acuity (BCVA) letter score using Early Treatment Diabetic Retinopathy Study (ETDRS) charts

  over a 24-week time period

• Change from Baseline in Best-corrected visual acuity (BCVA) letter score using Early Treatment Diabetic Retinopathy Study (ETDRS) charts

  over a 48-week time period

• Change from Baseline in lowest passing light level using Ora-VNC™ mobility test

  over a 24-week time period

Study Arms (1)

Single arm dose escalation study of VP-001

EXPERIMENTAL

Drug: VP-001

Interventions

VP-001 DRUG

Phase 1 open-label, single arm dose escalation study of VP-001 in participants with genetically confirmed PRPF31 mutation-associated retinal dystrophy

Single arm dose escalation study of VP-001

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 Baseline (Visit 2).
• Have a molecular (genetic) diagnosis of PRPF31 mutation.
• If ≥ 18 years of age, understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures. Are willing to comply with the instructions and attend all scheduled study visits.
• \. Have light perception (LP) or better vision in the study eye. 7. Participants of childbearing potential and male participants must not be pregnant or lactating and must be sexually inactive by abstinence, which is consistent with the preferred and usual lifestyle of the participant or agree to use adequate birth control throughout study duration. Adequate birth control is defined as hormonal - oral, implantable, injectable, or transdermal contraceptives; mechanical - spermicide in conjunction with a barrier such as a condom or diaphragm; intrauterine device (IUD); or surgical sterilization of partner. For nonsexually active participants, abstinence may be regarded as an adequate method of birth control. Participants of childbearing potential include all participants who have experienced menarche and have not undergone successful surgical sterilization (bilateral tubal ligation, hysterectomy, or bilateral oophorectomy) or are not post-menopausal (12 months after last menses).

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.
• 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-vascular endothelial growth factor (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 (scleral buckle, pars plana vitrectomy, retrieval of a dropped nucleus or intraocular lens, radial optic neurotomy, sheathotomy, cyclodestructive procedures or multiple filtration surgeries \[2 or more\]) 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 of Baseline (Visit 2), whichever is longer, or plan to participate in another study of drug or device during the study period. Participation in observational trials is allowable based on investigator discretion and consultation with the Medical Monitor. It is assumed that the observational trial evaluations would not interfere with participation in this study.
• Have received any prior cell or gene therapy for a retinal condition.
• Have a recent history (\<6 months) or current excessive recreational drug or alcohol use, in the opinion of the investigator.
• Any retinal pathology other than RP11 that in the investigator's opinion could affect study results.
• Participants should not have any conditions, in the investigator's opinion, that may put the participant at increased risk, confound study data, or interfere significantly with the participant's study participation.

Sponsors & Collaborators

• PYC Therapeutics: lead

Study Sites (6)

University of Florida Health

Jacksonville, Florida, 32209, United States

Location

Bascom Palmer Eye Institute University of Miami

Miami, Florida, 33136, United States

Location

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Oregon Health and Science University - Casey Eye Institute

Portland, Oregon, 97239, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75321, United States

Location

Baylor College of Medicine- Alkek Eye Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Retinal Dystrophies

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Sreenivasu Mudumba

  PYC

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 25, 2023
• First Posted: June 15, 2023
• Study Start: April 20, 2023
• Primary Completion: October 8, 2025
• Study Completion: October 8, 2025
• Last Updated: April 13, 2026

Record last verified: 2026-04

Locations

US (6)