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

NCT07058077 | Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: 0616-029U1111-1314-57962024-519068-42-00
• Study Type: interventional
• Target: 153
• Locations: 12 countries
• Sites: 25

Brief 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.

Conditions

Heterozygous Familial Hypercholesterolemia (HeFH)

Outcome Measures

Primary Outcomes (5)

• Part A: Maximum Plasma Concentration (Cmax) of Enlicitide

  Blood samples will be collected to determine the Cmax of enlicitide.

  At designated timepoints (up to 24 hours postdose on day 14)

• 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.

  At designated timepoints (up to 24 hours postdose on day 14)

• 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.

  Baseline and Week 24

• 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.

  Up to approximately 188 weeks

• 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.

  Up to approximately 180 weeks

Secondary Outcomes (7)

• Part B: Percent Change from Baseline in Apolipoprotein B (ApoB)

  Baseline and week 24

• Part B: Percent Change from Baseline in Non-High-Density Lipoprotein Cholesterol (non-HDL-C)

  Baseline and week 24

• Part B: Percent Change from Baseline in Lipoprotein (a) (Lp(a))

  Baseline and week 24

• Part B: Percentage of Participants With LDL-C <130 mg/dL at Week 24

  Week 24

• Part B: Percentage of Participants With ≥50% LDL-C LDL-C Reduction from Baseline at Week 24

  Baseline and week 24

Study Arms (4)

Part A: Enlicitide Decanoate

EXPERIMENTAL

Participants receive enlicitide decanoate orally once daily (QD) at a dosage determined by age for up to 2 weeks.

Drug: Enlicitide Decanoate

Part B: Enlicitide Decanoate

EXPERIMENTAL

Participants receive enlicitide decanoate QD at a dosage determined by age for up to 24 weeks.

Drug: Enlicitide Decanoate

Part B: Placebo

PLACEBO COMPARATOR

Participants receive placebo orally QD for up to 24 weeks.

Drug: Placebo

Open-Label Extension: Enlicitide Decanoate

EXPERIMENTAL

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

Interventions

Enlicitide Decanoate DRUG

Enlicitide decanoate taken by mouth

Also known as: MK-0616, Enlicitide

Open-Label Extension: Enlicitide Decanoate; Part A: Enlicitide Decanoate; Part B: Enlicitide Decanoate

Placebo DRUG

Placebo tablet matched to enlicitide decanoate taken by mouth

Part B: Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Merck Sharp & Dohme LLC: lead

Study Sites (25)

Nemours/Alfred I. duPont Hospital for Children ( Site 0001)

Wilmington, Delaware, 19803, United States

RECRUITING

Children's National Medical Center ( Site 0015)

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Excel Medical Clinical Trials ( Site 0008)

Boca Raton, Florida, 33434, United States

RECRUITING

Children's Healthcare of Atlanta Cardiology ( Site 0026)

Atlanta, Georgia, 30329, United States

RECRUITING

Cincinnati Children's Hospital Medical Center ( Site 0016)

Cincinnati, Ohio, 45229, United States

RECRUITING

West Virginia University ( Site 0013)

Morgantown, West Virginia, 26506, United States

RECRUITING

Monash Children s Hospital ( Site 1603)

Clayton, Victoria, 3168, Australia

RECRUITING

UZ Antwerpen ( Site 0601)

Edegem, Antwerpen, 2650, Belgium

RECRUITING

Universidade Federal Do Ceara ( Site 0201)

Fortaleza, Ceará, 60430270, Brazil

RECRUITING

Incor - Instituto do Coracao ( Site 0200)

São Paulo, 05403900, Brazil

RECRUITING

Beijing Anzhen Hospital. Capital Medical University ( Site 1917)

Beijing, Beijing Municipality, 100029, China

RECRUITING

Shanghai Children's Medical Center ( Site 1918)

Shanghai, Shanghai Municipality, 200120, China

RECRUITING

The Children's Hospital of Zhejiang University School of Medicine ( Site 1905)

Hangzhou, Zhejiang, 310057, China

RECRUITING

Clinica de la Costa S.A.S. ( Site 0400)

Barranquilla, Atlántico, 080020, Colombia

RECRUITING

Oncomédica S.A.S ( Site 0401)

Montería, Departamento de Córdoba, 230002, Colombia

RECRUITING

Fundación Cardiovascular de Colombia ( Site 0402)

Piedecuesta, Santander Department, 681017, Colombia

RECRUITING

Fundacion Valle del Lili ( Site 0403)

Cali, Valle del Cauca Department, 760032, Colombia

RECRUITING

New Childrens Hospital ( Site 0800)

Helsinki, Uusimaa, 00029, Finland

RECRUITING

Amsterdam UMC, locatie AMC ( Site 1000)

Amsterdam, North Holland, 1105 AZ, Netherlands

RECRUITING

New Zealand Clinical Research (Christchurch) ( Site 1700)

Christchurch, Canterbury, 8011, New Zealand

RECRUITING

National University Hospital-Paediatrics ( Site 1800)

Singapore, Central Singapore, 117599, Singapore

RECRUITING

Hospital Universitario Central de Asturias ( Site 1303)

Oviedo, Asturias, Principado de, 33011, Spain

RECRUITING

Hospital Clinico Universitario de Santiago de Compostela ( Site 1300)

Santiago de Compostela, La Coruna, 15706, Spain

RECRUITING

COMPLEJO HOSPITALARIO DE NAVARRA ( Site 1302)

Pamplona, Navarre, 31009, Spain

RECRUITING

Sheffield Childrens Hospital ( Site 1503)

Sheffield, S10 2TH, United Kingdom

RECRUITING

Related Links

• Merck Clinical Trials Information

MeSH Terms

Interventions

MK-0616

Study Officials

• Medical Director

  Merck Sharp & Dohme LLC

  STUDY DIRECTOR

Central Study Contacts

Toll Free Number

CONTACT

1-888-577-8839; Trialsites@msd.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Part A and the extension period are open-label. Part B is double-blinded.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: There are two parts of the study. Part A is a non randomized single treatment group. Part B is randomized parallel treatment groups. Participants who complete either part are then eligible to enroll in an open label extension (single group).

Study Record Dates

• First Submitted: July 1, 2025
• First Posted: July 10, 2025
• Study Start: August 21, 2025
• Primary Completion (Estimated): December 4, 2033
• Study Completion (Estimated): January 23, 2037
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

ES (3); GB (1); NL (1); BR (2); BE (1); CN (3); US (6); CO (4); NZ (1); FI (1); AU (1); SG (1)