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Enlicitide decanoate (MK-0616), an oral PCSK9 inhibitor, for children and adolescents with heterozygous familial hypercholesterolemia

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

Phase2; Phase3 Interventional Merck Sharp & Dohme LLC · NCT07058077

This trial tests whether a daily oral medicine called enlicitide decanoate can safely lower LDL cholesterol in children and teens with heterozygous familial hypercholesterolemia.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment153 (estimated)
Ages6 Years to 17 Years
SexAll
SponsorMerck Sharp & Dohme LLC Industry-sponsored
Locations26 sites (Wilmington, Delaware and 25 other locations)
Trial IDNCT07058077 on ClinicalTrials.gov

What this trial studies

This randomized, placebo-controlled Phase 2/3 interventional study gives children and adolescents with HeFH either oral enlicitide decanoate or placebo while monitoring safety, tolerability, and LDL-C changes. Participants must have a fasting LDL-C ≥130 mg/dL and be on stable background lipid-lowering therapy or have documented statin intolerance or refusal. The study includes pharmacokinetic sampling to see how the drug behaves in a child's body and regular lab and clinical assessments to track side effects. The primary clinical aim is to compare LDL-C lowering versus placebo while characterizing safety in this pediatric population.

Who should consider this trial

Good fit: Children and adolescents with a definite or possible diagnosis of HeFH, a fasting LDL-C ≥130 mg/dL, and who are on a stable lipid-lowering regimen or are statin-intolerant/refuse statins.

Not a fit: Patients with homozygous, compound, or double heterozygous FH, active nephrotic syndrome, significant malabsorption, or other listed exclusion conditions are unlikely to benefit from this study.

Why it matters

Potential benefit: If successful, the drug could provide an easy-to-take oral option that lowers LDL cholesterol in children and may reduce long-term cardiovascular risk.

How similar studies have performed: Injectable PCSK9 monoclonal antibodies have successfully lowered LDL-C in adults and some pediatric reports, but oral PCSK9 inhibitors like enlicitide are a newer approach with limited prior pediatric data.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Wilmington, Delaware and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Heterozygous Familial Hypercholesterolemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.