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

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified March 2026 by Merck Sharp & Dohme LLC
Sponsor
Merck Sharp & Dohme LLC
Information Provided by (Responsible Party)
Merck Sharp & Dohme LLC
Clinicaltrials.gov Identifier
NCT07058077
Other Study ID Numbers:
0616-029
First Submitted
June 30, 2025
First Posted
July 9, 2025
Last Update Posted
April 22, 2026
Last Verified
March 2026

ClinicalTrials.gov processed this data on April 2026. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Heterozygous Familial Hypercholesterolemia (HeFH)
Drug: Enlicitide DecanoateDrug: Enlicitide DecanoateDrug: PlaceboDrug: Enlicitide Decanoate

Study Design

Study TypeInterventional
Actual Enrollment153 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingQuadruple
Primary PurposeTreatment
Official TitleAn Operationally Seamless Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Pediatric Participants With Heterozygous Familial Hypercholesterolemia
Study Start DateAugust 20, 2025
Actual Primary Completion Date7yrs 7mos from now
Actual Study Completion Date10yrs 8mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
Part A: Enlicitide Decanoate
Participants receive enlicitide decanoate orally once daily (QD) at a dosage determined by age for up to 2 weeks.
Drug: Enlicitide Decanoate
Enlicitide decanoate taken by mouth
Part B: Enlicitide Decanoate
Participants receive enlicitide decanoate QD at a dosage determined by age for up to 24 weeks.
Drug: Enlicitide Decanoate
Enlicitide decanoate taken by mouth
Part B: Placebo
Participants receive placebo orally QD for up to 24 weeks.
Drug: Placebo
Placebo tablet matched to enlicitide decanoate taken by mouth
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.
Drug: Enlicitide Decanoate
Enlicitide decanoate taken by mouth

Outcome Measures

Primary Outcome Measures
  1. Part A: Maximum Plasma Concentration (Cmax) of Enlicitide
    Blood samples will be collected to determine the Cmax of enlicitide.
  2. Part A: Area Under the Concentration-Time Curve from 0 to 24 Hours (AUC0-24) of Enlicitide
    Blood samples will be collected to determine the AUC0-24 of enlicitide.
  3. Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C)
    Blood samples will be collected to determine the percent change from baseline in LDL-C.
  4. Number of Participants Who Experience an Adverse Event (AE)
    An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
  5. Number of Participants Who Discontinue Study Treatment Due to an AE
    An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Secondary Outcome Measures
  1. Part B: Percent Change from Baseline in Apolipoprotein B (ApoB)
    Blood samples will be collected to determine the percent change from baseline in apolipoprotein B.
  2. Part B: Percent Change from Baseline in Non-High-Density Lipoprotein Cholesterol (non-HDL-C)
    Blood samples will be collected to determine the percent change from baseline in non-HDL-C.
  3. Part B: Percent Change from Baseline in Lipoprotein (a) (Lp(a))
    Blood samples will be collected to determine the percent change from baseline in Lp(a).
  4. Part B: Percentage of Participants With LDL-C <130 mg/dL at Week 24
    The percentage of participants with LDL-C \&lt;130 mg/dL at week 24 will be reported.
  5. Part B: Percentage of Participants With ≥50% LDL-C LDL-C Reduction from Baseline at Week 24
    The percentage of participants with &ge;50% LDL-C reduction from baseline at week 24 will be reported.
  6. Part B: Percentage of Participants With LDL-C <100 mg/dL at Week 24
    The percentage of participants with LDL-C \&lt;100 mg/dL at week 24 will be reported.
  7. Change in Carotid Intima-media Thickness (cIMT)
    Ultrasound measurements will be performed to determine the change from baseline in cIMT.

Eligibility Criteria

Ages Eligible for Study(Child)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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

Contacts and Locations

Sponsors and CollaboratorsMerck Sharp & Dohme LLC
Locations
Nemours/Alfred I. duPont Hospital for Children ( Site 0001) | Wilmington Delaware, United States, 19803Children's National Medical Center ( Site 0015) | Washington D.C. District of Columbia, United States, 20010Excel Medical Clinical Trials ( Site 0008) | Boca Raton Florida, United States, 33434Children's Healthcare of Atlanta Cardiology ( Site 0026) | Atlanta Georgia, United States, 30329West Virginia University ( Site 0013) | Morgantown West Virginia, United States, 26506Monash Children s Hospital ( Site 1603) | Clayton Victoria, Australia, 3168UZ Antwerpen ( Site 0601) | Edegem Antwerpen, Belgium, 2650Universidade Federal Do Ceara ( Site 0201) | Fortaleza Ceará, Brazil, 60430270Incor - Instituto do Coracao ( Site 0200) | São Paulo , Brazil, 05403900Beijing Anzhen Hospital. Capital Medical University ( Site 1917) | Beijing Beijing Municipality, China, 100029Shanghai Children's Medical Center ( Site 1918) | Shanghai Shanghai Municipality, China, 200120The Children's Hospital of Zhejiang University School of Medicine ( Site 1905) | Hangzhou Zhejiang, China, 310057Clinica de la Costa S.A.S. ( Site 0400) | Barranquilla Atlántico, Colombia, 080020Oncomédica S.A.S ( Site 0401) | Montería Departamento de Córdoba, Colombia, 230002Fundación Cardiovascular de Colombia ( Site 0402) | Piedecuesta Santander Department, Colombia, 681017Fundacion Valle del Lili ( Site 0403) | Cali Valle del Cauca Department, Colombia, 760032New Childrens Hospital ( Site 0800) | Helsinki Uusimaa, Finland, 00029Amsterdam UMC, locatie AMC ( Site 1000) | Amsterdam North Holland, Netherlands, 1105 AZNew Zealand Clinical Research (Christchurch) ( Site 1700) | Christchurch Canterbury, New Zealand, 8011National University Hospital-Paediatrics ( Site 1800) | Singapore Central Singapore, Singapore, 117599Hospital Universitario Central de Asturias ( Site 1303) | Oviedo Asturias, Principado de, Spain, 33011Hospital Clinico Universitario de Santiago de Compostela ( Site 1300) | Santiago de Compostela La Coruna, Spain, 15706COMPLEJO HOSPITALARIO DE NAVARRA ( Site 1302) | Pamplona Navarre, Spain, 31009Sheffield Childrens Hospital ( Site 1503) | Sheffield , United Kingdom, S10 2TH
Investigators
Study Director: Medical Director, Merck Sharp & Dohme LLC