An Operationally Seamless Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

What this Trial Is About

This research aims to evaluate the safety and effectiveness of enlicitide decanoate in treating children and adolescents aged 6 to 17 years with heterozygous familial hypercholesterolemia (HeFH) who have high levels of low-density lipoprotein cholesterol (LDL-C). The study seeks to understand how children tolerate enlicitide, how the drug behaves in their bodies over time, and whether it lowers cholesterol levels better than a placebo. Participants will receive enlicitide decanoate orally once daily at doses adjusted by age during different study parts. Initially, treatment lasts up to 2 weeks, followed by a longer period of up to 24 weeks comparing enlicitide to a placebo. After completing these parts, eligible participants may join an open-label extension, receiving enlicitide daily for up to 3 years. Throughout the study, participants will undergo blood tests to measure cholesterol levels and drug concentration, as well as monitoring for any side effects. Researchers will track changes in LDL-C and other cholesterol markers, along with safety events, over periods ranging from two weeks up to several years. This includes evaluating the maximum drug concentration, cholesterol reductions at 24 weeks, and long-term safety up to approximately 3.5 years of follow-up.

A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)