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CompletedNCT05902962PlatypusUpdated May 29, 2026

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

A Phase 1 interventional study of VP-001 in Retinal Dystrophy, PRPF31 Mutationassociated Retinal Dystrophy and RP11, sponsored by PYC Therapeutics. Completed at 6 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-05-29.

Sponsored by PYC Therapeutics · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Aug 2025, 1 year 2 months ago, and no results have been posted to the registry.
Phase
Phase 1
Study type
Interventional
Enrollment
17
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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

02

Conditions studied

  • Retinal Dystrophy
  • PRPF31 Mutationassociated Retinal Dystrophy
  • RP11

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03

In context

Retinal Dystrophies

56 studies on the registry are indexed under Retinal Dystrophies; 16 are open to participants now.

This study's enrollment of 17 is below the median of 21 across 32 interventional studies indexed under Retinal Dystrophies.

Browse Retinal Dystrophies studies →

Lead sponsor

PYC Therapeutics is the lead sponsor of 8 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Male or female sex; ≥ 18 years of age at Baseline (Visit 2).
  2. Have a molecular (genetic) diagnosis of PRPF31 mutation.
  3. Have a clinical diagnosis of PRPF31 mutation-associated retinal dystrophy, that is, RP11. The following conditions are allowed for inclusion if due to RP11, if in the opinion of the investigator they will not interfere with study evaluations or have resolved: macular edema (intraretinal, sub-retinal or other fluid) requiring regular treatment at a frequency of less than every 6 weeks; macular edema must be stable for at least 3 months prior to Screening (Visit 1). The investigator must consult with the study Medical Monitor.
  4. 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.

6. 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).

Exclusion criteria

Exclusion Criteria:

  1. 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.
  2. 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.
  3. Have used anti-vascular endothelial growth factor (VEGF) agents within 2 months or corticosteroid injections within the last 3 months.
  4. Have had Ozurdex® implants placed within 3 months or Retisert® or Iluvien® implants placed within 3 years prior to Baseline (Visit 2).
  5. 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.
  6. Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the investigator.
  7. 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.
  8. Have received any prior cell or gene therapy for a retinal condition.
  9. Have a recent history (\<6 months) or current excessive recreational drug or alcohol use, in the opinion of the investigator.
  10. Any retinal pathology other than RP11 that in the investigator's opinion could affect study results.
  11. 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.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
17 participants (actual)

Study arms

  • Experimental
    Single arm dose escalation study of VP-001

    Drug: VP-001

Interventions

  • DrugVP-001

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

06

What researchers measure

Primary outcomes

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

    Time frame: over a 24-week time period

  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

    Time frame: over a 48-week time period

Secondary outcomes

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

    Time frame: over a 24-week time period

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

    Time frame: over a 48-week time period

  3. Incidence, severity relatedness and of non-ocular TEAEs

    Time frame: over a 24-week time period

  4. Incidence, severity relatedness and of non-ocular TEAEs

    Time frame: over a 48-week time period

Other outcomes

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

    Time frame: over a 24-week time period

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

    Time frame: over a 48-week time period

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

    Time frame: over a 24-week time period

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

    Time frame: over a 48-week time period

  5. Change from Baseline in Low luminance visual acuity (LLVA) letter score

    Time frame: over a 24-week time period

  6. Change from Baseline in Low luminance visual acuity (LLVA) letter score

    Time frame: over a 48-week time period

  7. Change from Baseline in Visual field sensitivity as measured by static perimetry with topographic analysis (Hill of Vision)

    Time frame: over a 24-week time period

  8. Change from Baseline in Visual field sensitivity as measured by static perimetry with topographic analysis (Hill of Vision)

    Time frame: over a 48-week time period

  9. Change from Baseline in Mean retinal sensitivity as measured by fundus-guided microperimetry

    Time frame: over a 24-week time period

  10. Change from Baseline in Mean retinal sensitivity as measured by fundus-guided microperimetry

    Time frame: over a 48-week time period

  11. Change from Baseline in Visual fields as measured by kinetic perimetry, utilizing I4e, III4e and V4e stimuli

    Time frame: over a 24-week time period

  12. Change from Baseline in Visual fields as measured by kinetic perimetry, utilizing I4e, III4e and V4e stimuli

    Time frame: over a 48-week time period

  13. Change from Baseline in Rod- and cone-mediated retinal function as measured by white, red and blue FST

    Time frame: over a 24-week time period

  14. Change from Baseline in Rod- and cone-mediated retinal function as measured by white, red and blue FST

    Time frame: over a 48-week time period

  15. Change from Baseline in Retinal thickness on SD-OCT, including retinal thickness in each ETDRS subfield and ellipsoid zone (EZ) area and volume

    Time frame: over a 24-week time period

  16. Change from Baseline in Retinal thickness on SD-OCT, including retinal thickness in each ETDRS subfield and ellipsoid zone (EZ) area and volume

    Time frame: over a 48-week time period

  17. Change from Baseline in Participant reported outcome measures utilizing the Patient Global Impressions of Change (PGI-C) and Patient Global Impressions of Severity (PGI-S)

    Time frame: over a 24-week time period

  18. Change from Baseline in Participant reported outcome measures utilizing the Patient Global Impressions of Change (PGI-C) and Patient Global Impressions of Severity (PGI-S)

    Time frame: over a 48-week time period

  19. Change from Baseline in Retinal function using full-field electroretinography (ERG)

    Time frame: over a 24-week time period

  20. Change from Baseline in Retinal function using full-field electroretinography (ERG)

    Time frame: over a 48-week time period

  21. Change from Baseline in Area of hypo-autofluorescence captured by FAF

    Time frame: over a 24-week time period

  22. Change from Baseline in Area of hypo-autofluorescence captured by FAF

    Time frame: over a 48-week time period

  23. Change from Baseline in Abnormalities captured by wide-field fundus photography

    Time frame: over a 24-week time period

  24. Change from Baseline in Abnormalities captured by wide-field fundus photography

    Time frame: over a 48-week time period

07

Study locations

6 sites
  • University of Florida Health
    Jacksonville, Florida 32209, United States
  • Bascom Palmer Eye Institute University of Miami
    Miami, Florida 33136, United States
  • University of Michigan Kellogg Eye Center
    Ann Arbor, Michigan 48105, United States
  • Oregon Health and Science University - Casey Eye Institute
    Portland, Oregon 97239, United States
  • Retina Foundation of the Southwest
    Dallas, Texas 75321, United States
  • Baylor College of Medicine- Alkek Eye Center
    Houston, Texas 77030, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 29, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT05902962
Lead sponsor
PYC Therapeutics
Responsible party
Sponsor
First posted
Jun 15, 2023
Start date
Apr 20, 2023
Primary completion
Aug 8, 2025
Completion
Aug 8, 2025
Last update
May 29, 2026

Study contacts

Sreenivasu Mudumba
study chair · PYC

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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