VP-001 for Retinal Dystrophy
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, to determine its safety and tolerability for individuals with retinal dystrophy linked to the PRPF31 gene mutation. Retinal dystrophy can cause vision problems, so the study aims to explore whether VP-001 can help manage these issues. The trial seeks participants diagnosed with PRPF31-related retinal dystrophy who have specific levels of vision impairment. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Do I have to stop taking my current medications for the trial?
The trial does not specify if you need to stop taking your current medications, but you cannot have used anti-VEGF agents within 2 months or corticosteroid injections within the last 3 months. It's best to discuss your specific medications with the study team.
Is there any evidence suggesting that VP-001 is likely to be safe for humans?
Research has shown that VP-001 was well-tolerated in earlier studies. In one study, participants with a specific genetic condition received this treatment multiple times, and it proved to be safe. They did not experience any major side effects, indicating no serious or unexpected problems. This suggests that VP-001 is generally safe for humans based on existing data. However, since the current trial is in an early stage, the main goal is to further confirm its safety and assess how well people can tolerate it.12345
Why do researchers think this study treatment might be promising?
VP-001 is unique because it offers a novel approach to treating retinal dystrophy. Unlike existing treatments, which often focus on managing symptoms with vitamins or surgery, VP-001 targets the underlying causes of the condition at a molecular level. Researchers are excited about VP-001 because it uses a new mechanism of action that could potentially stop or even reverse the progression of retinal dystrophy. This innovative approach provides hope for more effective and long-lasting results compared to current therapies.
What evidence suggests that VP-001 might be an effective treatment for retinal dystrophy?
Research has shown that VP-001, the investigational treatment in this trial, offers promising results for treating retinal dystrophy caused by the PRPF31 mutation. Studies have found that VP-001 can enhance visual clarity. In one study, participants achieved better vision scores, improving from seeing at 20/47 to a clearer level. The treatment also appears to slow the loss of cells in the eye that are crucial for vision. Overall, these findings suggest that VP-001 may effectively aid individuals with this specific type of retinal dystrophy.23467
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive multiple ascending doses of intravitreally administered VP-001
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