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Virus Therapy

VP-001 for Retinal Dystrophy

Phase 1
Waitlist Available
Research Sponsored by PYC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Have a molecular (genetic) diagnosis of PRPF31 mutation
Meets visual function criteria in the study eye
Must not have
Recent vitreoretinal surgery
Mutations in genes causing specific types of retinitis pigmentosa
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This trial is testing the safety of a new treatment called VP-001 in people with a specific type of retinal dystrophy.

Who is the study for?
This trial is for individuals with confirmed PRPF31 mutation-associated retinal dystrophy, a genetic disorder causing vision loss. Details on specific inclusion and exclusion criteria are not provided, but typically participants must meet certain health standards and not have conditions that could interfere with the study.
What is being tested?
The trial is testing multiple increasing doses of VP-001 given through an injection into the eye (intravitreally) to assess its safety and how well it's tolerated in patients with retinal dystrophy due to PRPF31 mutations.
What are the potential side effects?
Specific side effects of VP-001 are not listed; however, as this is a Phase 1 trial focusing on safety, potential side effects may include eye irritation or discomfort, inflammation, changes in vision, or other localized reactions at the site of injection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a PRPF31 genetic mutation.
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My vision in the study eye meets the required standards.
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I have been diagnosed with a specific genetic eye condition (RP11).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had eye surgery recently.
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My retinitis pigmentosa is caused by a specific genetic mutation.
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I have cloudiness or issues with my eye that affects vision.
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I have had cell, RNA, or gene therapy for an eye condition.
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My eye condition is not RP11.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

1Treatment groups
Experimental Treatment
Group I: Multi-ascending dose escalation study of VP-001Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

PYC TherapeuticsLead Sponsor
4 Previous Clinical Trials
128 Total Patients Enrolled
~8 spots leftby Nov 2025
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