VP-001 for Retinal Dystrophy
(Platypus Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called VP-001, a potential drug for retinal dystrophy caused by a PRPF31 gene mutation. The main goal is to determine if VP-001 is safe and tolerable for individuals with this genetic form of vision loss. Participants must have a diagnosis of this condition, which affects sight, and retain some vision in at least one eye. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.
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 VP-001 is likely to be safe for humans?
Studies have shown that VP-001, administered as an eye injection, is generally safe. In earlier tests, participants did not experience major side effects. Some reported mild discomfort, such as redness or irritation, but these effects were temporary. Research indicates that the treatment is well-tolerated. Although this study is in the early stages with limited data, the main goal is to ensure the treatment's safety. Prospective participants should remember that the primary focus is on assessing safety first.12345
Why do researchers think this study treatment might be promising?
VP-001 is unique because it offers a new approach for treating retinal dystrophy, potentially addressing the root causes of the condition rather than just managing symptoms. Unlike current treatments that may focus on slowing disease progression, VP-001 targets specific genetic factors involved in retinal degeneration, which could lead to more effective and lasting results. Researchers are excited about VP-001 because it could represent a significant advancement in precision medicine for eye disorders, offering hope for improved vision outcomes for patients with retinal dystrophy.
What evidence suggests that VP-001 might be an effective treatment for retinal dystrophy?
Research shows that VP-001 can improve vision in people with an eye condition caused by the PRPF31 gene mutation. Previous studies have found that this treatment makes it easier for patients to see clearly. In one study, vision scores improved from about 20/47 to a better score. This suggests that VP-001 might slow down or even reverse some vision loss related to this condition. While more research is needed, these early results are promising and suggest that VP-001 could be effective.25678
Who Is on the Research Team?
Sreenivasu Mudumba
Principal Investigator
PYC
Are You a Good Fit for This Trial?
Adults with a genetic diagnosis of PRPF31 mutation-associated retinal dystrophy (RP11) can join this trial. They must be willing to follow the study plan and attend all visits, use effective birth control if they can have children, and not be pregnant or breastfeeding. People with uncontrolled diseases, recent certain eye treatments or surgeries, other gene therapy for retinal conditions, or excessive drug/alcohol use cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive intravitreal administration of VP-001 in a dose escalation manner to evaluate safety and tolerability
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