Clinical trial of enlicitide in children and adolescents with familial high cholesterol

• Research type

  Research Study

• Full title

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

• IRAS ID

  1012479

• Contact name

  - -

• Contact email

  N/A

• Sponsor organisation

  Merck Sharp & Dohme LLC

• Clinicaltrials.gov Identifier

  NCT07058077

• Research summary

  Researchers are looking for new ways to treat children with heterozygous familial hypercholesterolaemia (HeFH). HeFH causes high amounts of low-density lipoprotein cholesterol (LDL-C) in the blood. Too much LDL-C can cause fatty deposits to stick to the walls of arteries causing them to narrow, meaning less blood can flow through. This can lead to major heart-related events.

  Enlicitide, the trial medicine (also known as enlicitide decanoate or MK-0616), is designed to reduce LDL-C and is taken by mouth as a tablet.

  The goals are to learn:
  • About the safety of enlicitide and if children tolerate it
  • What happens to enlicitide in a child’s body over time
  • If enlicitide works to lower cholesterol levels in children more than placebo

  About 153 children with HeFH will be in this trial. Children can be in Part A or Part B (not both) if they:
  • Are 6 years old to less than 18 years old at the start of the trial (Group 1: 12 years old to less than 18 years old and Group 2: 6 years old to less than 12 years old)
  • Currently take medicines to lower high cholesterol
  • Can swallow tablets
  • Do not have homozygous familial hypercholesterolaemia

  Part A (Open-label, about 3.5 months): Children will take enlicitide for 2 weeks. Researchers will check for safety concerns and the amount of enlicitide in the blood in Part A before starting Part B for that age group.
  Part B (Double-blind, about 9 months): Researchers will randomly assign children by chance to receive enlicitide (2 out of 3 chance) or placebo (1 out of 3 chance). Children will take their assigned trial medicine for about 6 months.
  Open-label Extension: Children who finish Part A or B will be offered the chance to continue taking enlicitide for up to 3 years.

  Children may:
  • Give urine and blood samples
  • Have tests to check the heart (electrocardiogram)
  • Have their cheek swabbed
  • Have exams to check their growth
  • Have ultrasounds to check the thickness of blood vessels

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  25/LO/0612

• Date of REC Opinion

  19 Nov 2025

• REC opinion

  Further Information Favourable Opinion