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Experimental eye drug shows promise for rare Blindness-Causing disease

NCT ID NCT06852963

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called VP-001 for people with a rare genetic eye disease that causes vision loss. The drug is injected into the eye and aims to slow or stop further damage. Researchers will check safety and measure any changes in eyesight over about two years. The trial includes 17 adults who have already received VP-001 in earlier studies.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

17 people

The number who actually took part.

Started

Jun 2025

Expected to finish

Apr 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Bascom Palmer Eye Institute - University of Miami

    Miami, Florida, 33136, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Casey Eye Institute - OHSU

    Portland, Oregon, 97239, United States

  • Kellogg Eye Center - University of Michigan

    Ann Arbor, Michigan, 48105, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231, United States

  • University of Florida College of Medicine

    Jacksonville, Florida, 32209, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.