VP-001 for Retinal Dystrophy
(DINGO Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety and effectiveness of VP-001, an experimental treatment for Retinal Dystrophy linked to a PRPF31 gene mutation. The trial examines various doses and schedules of VP-001 to assess its efficacy when administered multiple times directly into the eye. It targets individuals previously treated with VP-001 who have Retinitis Pigmentosa caused by the PRPF31 mutation. As a Phase 1, Phase 2 trial, this research aims to understand the treatment's function in people and measure its effectiveness in an initial, smaller group.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, you cannot have used anti-VEGF agents within 2 months or corticosteroid injections within the last 3 months before starting the trial.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that VP-001 has been generally well-tolerated in previous studies. One study found single injections of VP-001 at doses of 3 micrograms (µg) and 10 micrograms to be safe. Even a higher dose of 30 micrograms caused no major problems. Another report suggested that VP-001 appears safe for treating eye conditions.
These findings indicate that VP-001 is likely safe for humans. However, this trial involves higher doses and repeated treatments. Participants should consider these factors and discuss any concerns with the study team.12345Why are researchers excited about this trial's treatment?
Unlike the standard treatments for retinal dystrophy, which often include gene therapies or vitamin A supplementation, VP-001 offers a unique approach by using an intravitreal (IVT) delivery method to administer a specific dosage of the active compound directly into the eye. Researchers are excited about VP-001 because it targets the retina more precisely, potentially leading to improved outcomes and fewer systemic side effects. Additionally, the varying dosages and frequency of administration being tested, such as 75ug or 120ug every 8 or 12 weeks, provide flexibility and could optimize treatment effectiveness for different patients. This innovative approach could significantly enhance how retinal dystrophy is managed, offering hope for better vision preservation.
What evidence suggests that this trial's treatments could be effective for Retinal Dystrophy?
Research has shown that VP-001 yields promising results for treating retinal dystrophy caused by the PRPF31 mutation. In earlier studies, patients experienced significant vision improvement, measured by logMAR scores, which assess vision clarity. The scores improved from about 20/47 to a better level, indicating clearer vision. The treatment also slowed the loss of photoreceptor cells, crucial for sight. Overall, these findings suggest that VP-001 could effectively treat retinal dystrophy linked to the PRPF31 mutation.56789
Are You a Good Fit for This Trial?
This trial is for individuals with PRPF31 mutation-associated Retinal Dystrophy who have already been treated with VP-001. Specific eye conditions like fungal retinitis, Purtscher retinopathy, and other related diseases are the focus. Participants must meet certain health standards to be included.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive repeat doses of VP-001 intravitreally, with dosing regimens of either 75ug or 120ug every 8 or 12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- VP-001
Find a Clinic Near You
Who Is Running the Clinical Trial?
PYC Therapeutics
Lead Sponsor