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

Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 17
Healthy Volunteers: f
View:

• 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

Locations
United States
Delaware
Nemours/Alfred I. duPont Hospital for Children (0001)
RECRUITING
Wilmington
Florida
Excel Medical Clinical Trials (0008)
RECRUITING
Boca Raton
Contact Information
Primary
Toll Free Number
Trialsites@msd.com
1-888-577-8839
Time Frame
Start Date: 2025-08-21
Estimated Completion Date: 2037-01-24
Participants
Target number of participants: 153
Treatments
Experimental: Part A: Enlicitide Decanoate
Participants receive enlicitide decanoate orally once daily (QD) at a dosage determined by age for up to 2 weeks.
Experimental: Part B: Enlicitide Decanoate
Participants receive enlicitide decanoate QD at a dosage determined by age for up to 24 weeks.
Placebo_comparator: Part B: Placebo
Participants receive placebo orally QD for up to 24 weeks.
Experimental: Open-Label Extension: Enlicitide Decanoate
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.
Sponsors
Leads: Merck Sharp & Dohme LLC

This content was sourced from clinicaltrials.gov

Similar Clinical Trials