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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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)
  * A nonstatin LLT with documented intolerance to at least 2 different statins, or documented intolerance to 1 statin plus 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 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 it's running

• 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
• Boston Children's Hospital ( Site 0018)
  Boston, Massachusetts, United States Recruiting
• St. Louis Children's Hospital ( Site 0014)
  St Louis, Missouri, United States Recruiting

Study contacts

• Study coordinator: Toll Free Number
• Email: Trialsites@msd.com
• Phone: 1-888-577-8839

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• ORIGIN-FH: Newborn screening to detect familial high cholesterol

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• SYH2053 Injection for adults with heterozygous familial hypercholesterolemia

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• Coronary Plaque Progression in HeFH: DESTINY-FH

  This study tests how heart arteries change over 5 years in people with heterozygous familial hypercholesterolemia using coronary CT angiography.

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• Evaluating Inclisiran for Children with Familial Hypercholesterolemia

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• Detecting Familial Hypercholesterolemia in Children

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  Recruiting at 2 sites Institut Investigacio Sanitaria Pere Virgili · Reus, Tarragona and 1 other locations · NCT04370899

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.