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

Phase 3RecruitingNCT07058077

Merck Sharp & Dohme LLC153 enrolled

Overview

This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density lipoprotein cholesterol (LDL-C) in the blood. The goals of this study are to learn about the safety of enlicitide and if children tolerate it, what happens to enlicitide in a child's body over time, and if enlicitide works to lower cholesterol levels in children more than a placebo.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

153

Conditions

Heterozygous Familial Hypercholesterolemia (HeFH)

Interventions

Enlicitide Decanoate

Eligibility

Sex: ALLMin age: 6 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: Inclusion criteria include, but are not limited to: * Has possible or definite diagnosis of HeFH based on a locally accepted diagnostic algorithm or diagnosis by genetic testing results * Has a fasted LDL-C value (evaluated by the central laboratory) that is ≥130 mg/dL * Is receiving either an optimized daily dose of statin (± nonstatin LLT); or a nonstatin LLT with documented intolerance to at least 2 different statins or refusal of statin therapy by the participant or legally acceptable representative * Is on a stable dose of all background LLTs for at least 30 days prior to screening, with no medication or dose changes planned during participation in Part A or Part B Exclusion Criteria: Exclusion criteria include, but are not limited to: * Has a history of homozygous FH based on genetic or clinical criteria, or history of known compound heterozygous FH, or double heterozygous FH * Has a history of nephrotic syndrome * Has any clinically significant malabsorption condition based on principal investigator assessment * Was previously treated/is being treated with certain other cholesterol lowering medications, including proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors without adequate washout

Locations (13)

Nemours/Alfred I. duPont Hospital for Children ( Site 0001)

Wilmington, Delaware, United States

RECRUITING

Children's National Medical Center ( Site 0015)

Washington D.C., District of Columbia, United States

RECRUITING

Excel Medical Clinical Trials ( Site 0008)

Boca Raton, Florida, United States

RECRUITING

Children's Healthcare of Atlanta Cardiology ( Site 0026)

Atlanta, Georgia, United States

RECRUITING

Universidade Federal Do Ceara ( Site 0201)

Fortaleza, Ceará, Brazil

RECRUITING

Incor - Instituto do Coracao ( Site 0200)

São Paulo, Brazil

RECRUITING

Beijing Anzhen Hospital. Capital Medical University ( Site 1917)

Beijing, Beijing Municipality, China

RECRUITING

The Children's Hospital of Zhejiang University School of Medicine ( Site 1905)

Hangzhou, Zhejiang, China

RECRUITING

Clinica de la Costa S.A.S. ( Site 0400)

Barranquilla, Atlántico, Colombia

RECRUITING

Oncomédica S.A.S ( Site 0401)

Montería, Departamento de Córdoba, Colombia

RECRUITING

...and 3 more locations

Outcomes

Primary Outcomes

Part A: Maximum Plasma Concentration (Cmax) of Enlicitide

Blood samples will be collected to determine the Cmax of enlicitide.

Time frame: At designated timepoints (up to 24 hours postdose on day 14)

Part A: Area Under the Concentration-Time Curve from 0 to 24 Hours (AUC0-24) of Enlicitide

Blood samples will be collected to determine the AUC0-24 of enlicitide.

Time frame: At designated timepoints (up to 24 hours postdose on day 14)

Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C)

Blood samples will be collected to determine the percent change from baseline in LDL-C.

Time frame: Baseline and Week 24

Number of Participants Who Experience an Adverse Event (AE)

An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

Time frame: Up to approximately 188 weeks

Number of Participants Who Discontinue Study Treatment Due to an AE

An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

Time frame: Up to approximately 180 weeks