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

Merck Sharp & Dohme (MSD)

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Heterozygous Familial Hypercholesterolemia (HeFH)

Treatments: Drug: Placebo; Drug: Enlicitide Decanoate

Study type: Interventional

Funder types: Industry

Identifiers

NCT07058077

2024-519068-42-00 (Registry Identifier)

U1111-1314-5796 (Registry Identifier)

0616-029

Details and patient eligibility

About

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.

Enrollment: 153 estimated patients

Sex: All

Ages: 6 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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153 participants in 4 patient groups, including a placebo group

Part A: Enlicitide Decanoate

Experimental group

Description:

Participants receive enlicitide decanoate orally once daily (QD) at a dosage determined by age for up to 2 weeks.

Treatment:

Drug: Enlicitide Decanoate

Part B: Enlicitide Decanoate

Experimental group

Description:

Participants receive enlicitide decanoate QD at a dosage determined by age for up to 24 weeks.

Treatment:

Drug: Enlicitide Decanoate

Part B: Placebo

Placebo Comparator group

Description:

Participants receive placebo orally QD for up to 24 weeks.

Treatment:

Drug: Placebo

Open-Label Extension: Enlicitide Decanoate

Experimental group

Description:

Participants who complete either Part A or Part B may enroll in this open-label extension arm. Participants in the extension arm receive enlicitide decanoate QD at a dosage determined by age for up to 3 years.

Treatment:

Drug: Enlicitide Decanoate