An Operationally Seamless Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Pediatric Participants with Heterozygous Familial Hypercholesterolemia

Condition studied

​Heterozygous Familial Hypercholesterolemia​ in pediatric patients

Investigational medicinal product(s)

Eligibility

Primary endpoint

Maximum Plasma Concentration (Cmax) of Enlicitide for Part A (Age Cohort 1), Maximum Plasma Concentration (Cmax) of Enlicitide for Part A (Age Cohort 2), Area Under the Concentration-time Curve From 0 to 24 (AUC0-24) Hours of Enlicitide for Part A (Age Cohort 1), Area Under the Concentration-time Curve From 0 to 24 (AUC0-24) Hours of Enlicitide for Part A (Age Cohort 2), Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 in Part B Participants, Percent Change From Baseline in LDL-C at Week 24 in Part B Age Cohort 1 Participants, Number of Participants With One or More Adverse Events (AEs), Number of Participants Who Discontinue Study Treatment Due to an AE

Endpoint detail

Percentage Change From Baseline in Apolipoprotein B (ApoB) at Week 24 in Part B Participants, Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 in Part B Participants, Percentage Change From Baseline in Lipoprotein (a) [Lp (a)] at Week 24 in Part B Participants, Proportion of Participants With LDL-C <130 mg/dL (3.37 mmol/L) at Week 24 in Part B Participants, Proportion of Participants LDL-C ≥50% Reduction From Baseline at Week 24 in Part B Participants, Proportion of Participants With LDL-C <100 mg/dL (2.59 mmol/L) at Week 24 in Part B Participants, Change in Ultrasound Determined Mean cIMT at Week 24 in Part B Participants