VERVE-201 for High Cholesterol
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called VERVE-201, which aims to lower LDL cholesterol, often referred to as "bad" cholesterol. The trial targets individuals with refractory hypercholesterolemia, meaning their high cholesterol levels resist standard treatments. Participants will receive a single dose to assess the treatment's safety and its impact on cholesterol levels. Ideal candidates are those with high cholesterol unresponsive to other treatments and without liver disease. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot be on a monoclonal antibody targeting ANGPTL3 or have used one recently.
Is there any evidence suggesting that VERVE-201 is likely to be safe for humans?
Research has shown that VERVE-201 was well-tolerated in animal studies with primates. These studies noted only temporary increases in liver-related markers, a common occurrence with treatments using tiny fat particles to deliver drugs. No serious side effects linked to VERVE-201 were reported in these studies.
As this trial is a Phase 1 study, researchers primarily focus on assessing the treatment's safety in humans. This phase typically marks the first step in testing a new treatment in people. While results in animals have been promising, human trials are essential to fully understand how well people tolerate the treatment.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for high cholesterol, which typically include statins and other cholesterol-lowering medications, VERVE-201 is unique because it uses gene-editing technology to target the root cause of high cholesterol. While most treatments work by inhibiting cholesterol production or absorption, VERVE-201 aims to make permanent changes at the genetic level, potentially providing a long-lasting solution with a single dose. Researchers are excited about VERVE-201 because it could dramatically reduce the need for lifelong medication and improve patient adherence by offering a one-time treatment option.
What evidence suggests that VERVE-201 might be an effective treatment for high cholesterol?
Research shows that VERVE-201, which uses advanced gene-editing technology, may effectively lower LDL cholesterol levels. In early studies, treatments like VERVE-201 reduced LDL cholesterol by about 47%. This treatment targets and turns off the ANGPTL3 gene in the liver, crucial for controlling cholesterol. Initial results from similar research suggest it could be a promising option for managing high cholesterol, especially for patients who haven't succeeded with other treatments. Participants in this trial will receive a single dose of VERVE-201 as part of the dose escalation study.12356
Are You a Good Fit for This Trial?
This trial is for men and women who cannot bear children and have refractory hypercholesterolemia, which means their high cholesterol levels remain high despite treatment. Participants should not be able to get pregnant because the effects of VERVE-201 on childbearing are unknown.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single ascending dose of VERVE-201 to evaluate safety and pharmacodynamics
Follow-up
Participants are monitored for safety and effectiveness after receiving the single dose
What Are the Treatments Tested in This Trial?
Interventions
- VERVE-201
Find a Clinic Near You
Who Is Running the Clinical Trial?
Verve Therapeutics, Inc.
Lead Sponsor