Enlicitide Decanoate for High Cholesterol
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if the medication enlicitide decanoate, a new potential drug, is safe and effective for children and teens with heterozygous familial hypercholesterolemia (HeFH), a condition that causes high levels of bad cholesterol (LDL-C) in the blood. Researchers will assess whether this treatment lowers cholesterol levels more effectively than a placebo, a non-active substance. The trial consists of two parts: some participants will take the medicine for a few weeks, while others may continue for up to three years if they show progress. Children and teens with a confirmed HeFH diagnosis and high LDL-C levels, despite using other cholesterol-lowering medications, might be suitable candidates for this trial. As a Phase 2, Phase 3 trial, this study evaluates the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking research.
Do I have to stop taking my current medications for the trial?
You need to be on a stable dose of your current cholesterol medications for at least 30 days before the trial and cannot change them during the trial. If you are taking certain cholesterol medications like PCSK9 inhibitors, you may need a break from them before starting the trial.
Is there any evidence suggesting that enlicitide decanoate is likely to be safe for children and adolescents?
Research has shown that enlicitide decanoate is generally safe for adults with heterozygous familial hypercholesterolemia (HeFH), a genetic condition causing high cholesterol. Studies have found that enlicitide effectively lowers "bad" LDL cholesterol and has a safety profile similar to a placebo. This indicates that people taking the drug did not experience more side effects than those taking a sugar pill. The most common issues related to the treatment were mild, such as headaches and cold-like symptoms. These findings suggest that enlicitide decanoate could be a safe option for managing high cholesterol in children and teenagers.12345
Why do researchers think this study treatment might be promising for high cholesterol?
Unlike the standard treatments for high cholesterol, such as statins, Enlicitide Decanoate offers a novel approach with its unique mechanism of action. While statins work by blocking the enzyme responsible for cholesterol production in the liver, Enlicitide Decanoate targets cholesterol absorption directly, potentially making it effective for patients who don't respond well to existing therapies. Additionally, this treatment is taken orally once daily, which could offer a more convenient option for patients. Researchers are particularly excited about its potential to deliver quicker results, with some participants seeing benefits in just a few weeks.
What evidence suggests that enlicitide decanoate might be an effective treatment for high cholesterol?
Research has shown that enlicitide decanoate can significantly lower LDL-C, often called "bad cholesterol," in people with high cholesterol. One study found that enlicitide reduced LDL-C levels by about 47.6% over a year. This reduction was not only statistically significant but also important for health, potentially improving health outcomes. Enlicitide was generally well tolerated, with most people not experiencing serious side effects. These findings suggest that enlicitide may effectively manage high cholesterol, even in younger patients with HeFH. Participants in this trial will receive enlicitide decanoate at different stages, with some receiving it for up to 24 weeks and others having the option to continue in an open-label extension for up to 3 years.12367
Who Is on the Research Team?
Medical Director
Principal Investigator
Merck Sharp & Dohme LLC
Are You a Good Fit for This Trial?
This trial is for children and adolescents with a condition called heterozygous familial hypercholesterolemia (HeFH), which causes high cholesterol. Participants must have an LDL-C level of ≥130 mg/dL, be on stable cholesterol-lowering treatments, and can't start or change these medications during the study.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Participants receive enlicitide decanoate orally once daily at a dosage determined by age for up to 2 weeks
Treatment Part B
Participants receive enlicitide decanoate or placebo orally once daily at a dosage determined by age for up to 24 weeks
Open-label Extension
Participants who complete either Part A or Part B may enroll in this open-label extension arm and receive enlicitide decanoate for up to 3 years
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Enlicitide Decanoate
Find a Clinic Near You
Who Is Running the Clinical Trial?
Merck Sharp & Dohme LLC
Lead Sponsor
Chirfi Guindo
Merck Sharp & Dohme LLC
Chief Marketing Officer since 2022
Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business
Robert M. Davis
Merck Sharp & Dohme LLC
Chief Executive Officer since 2021
JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University