Experimental eye drug shows promise for rare Blindness-Causing disease
NCT ID NCT06852963
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.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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17 people
The number who actually took part.
- Started
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Jun 2025
- Expected to finish
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Apr 2028
An estimate. End dates often move.
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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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
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Bascom Palmer Eye Institute - University of Miami
Miami, Florida, 33136, United States
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Casey Eye Institute - OHSU
Portland, Oregon, 97239, United States
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Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, 48105, United States
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Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
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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.
- Can a contact lens improve clarity during retinal surgery?
- Can an eye injection slow a genetic cause of blindness?
- Can a single eye injection restore sight in genetic blindness?
- A sharper look at the retina: new imaging technology may spot eye disease earlier
- Stem cell eye transplant aims to halt blindness from rare genetic disease
- Could a smartphone camera spot blinding eye diseases?