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Recruiting NCT07058077

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

Phase II / Phase III Interventional Heterozygous Familial Hypercholesterolemia (HeFH)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Enlicitide Decanoate, Placebo.
Who it may be relevant to
Registry conditions: Heterozygous Familial Hypercholesterolemia (HeFH). Basic parameters: 6 years — 17 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Belgium, Brazil, Chile +11
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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.

Interventions

  • Drug Enlicitide Decanoate
    Enlicitide decanoate taken by mouth
  • Drug Placebo
    Placebo tablet matched to enlicitide decanoate taken by mouth

Primary outcome measures

  • Part A: Maximum Plasma Concentration (Cmax) of Enlicitide [Time frame: 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 [Time frame: At designated timepoints (up to 24 hours postdose on day 14)]
  • Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) [Time frame: Baseline and Week 24]
  • Number of Participants Who Experience an Adverse Event (AE) [Time frame: Up to approximately 188 weeks]
  • Number of Participants Who Discontinue Study Treatment Due to an AE [Time frame: Up to approximately 180 weeks]
Secondary outcome measures (7)
  • Part B: Percent Change from Baseline in Apolipoprotein B (ApoB) [Time frame: Baseline and week 24]
  • Part B: Percent Change from Baseline in Non-High-Density Lipoprotein Cholesterol (non-HDL-C) [Time frame: Baseline and week 24]
  • Part B: Percent Change from Baseline in Lipoprotein (a) (Lp(a)) [Time frame: Baseline and week 24]
  • Part B: Percentage of Participants With LDL-C <130 mg/dL at Week 24 [Time frame: Week 24]
  • Part B: Percentage of Participants With ≥50% LDL-C Reduction from Baseline at Week 24 [Time frame: Baseline and week 24]
  • Part B: Percentage of Participants With LDL-C <100 mg/dL at Week 24 [Time frame: Week 24]
  • Change in Carotid Intima-media Thickness (cIMT) [Time frame: Baseline and week 24]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 7 centers
  • Nemours/Alfred I. duPont Hospital for Children ( Site 0001) — Wilmington
  • Children's National Medical Center ( Site 0015) — Washington D.C.
  • Excel Medical Clinical Trials ( Site 0008) — Boca Raton
  • Children's Healthcare of Atlanta Cardiology ( Site 0026) — Atlanta
  • Boston Children's Hospital ( Site 0018) — Boston
  • Cincinnati Children's Hospital Medical Center ( Site 0016) — Cincinnati
  • West Virginia University ( Site 0013) — Morgantown
China · 5 centers
  • Beijing Anzhen Hospital. Capital Medical University ( Site 1917) — Beijing
  • Guangdong Provincial People's Hospital ( Site 1915) — Guangzhou
  • Children's Hospital of Fudan University ( Site 1906) — Shanghai
  • Shanghai Children's Medical Center ( Site 1918) — Shanghai
  • The Children's Hospital of Zhejiang University School of Medicine ( Site 1905) — Hangzhou
Colombia · 4 centers
  • Clinica de la Costa S.A.S. ( Site 0400) — Barranquilla
  • Oncomédica S.A.S ( Site 0401) — Montería
  • Fundación Cardiovascular de Colombia ( Site 0402) — Piedecuesta
  • Fundacion Valle del Lili ( Site 0403) — Cali
Brazil · 3 centers
  • Universidade Federal Do Ceara ( Site 0201) — Fortaleza
  • Instituto Dante Pazzanese de Cardiology ( Site 0206) — São Paulo
  • Incor - Instituto do Coracao ( Site 0200) — São Paulo
Mexico · 3 centers
  • Laboratorio de Patología Clínica del Hospital Universitario "Dr. José Eleuterio González" — Monterrey
  • Centro de Investigacion Clinica de Oaxaca ( Site 0505) — Oaxaca City
  • INVECORDIS S.C. ( Site 0503) — Huixquilucan
Spain · 3 centers
  • Hospital Universitario Central de Asturias ( Site 1303) — Oviedo
  • Hospital Clinico Universitario de Santiago de Compostela ( Site 1300) — Santiago de Compostela
  • COMPLEJO HOSPITALARIO DE NAVARRA ( Site 1302) — Pamplona
United Kingdom · 3 centers
  • Birmingham Childrens Hospital ( Site 1501) — Birmingham
  • Sheffield Childrens Hospital ( Site 1503) — Sheffield
  • Southampton General Hospital ( Site 1502) — Southampton
Chile · 2 centers
  • Explora Salud ( Site 0304) — Santiago
  • Pontificia Universidad Catolica de Chile ( Site 0300) — Santiago
Australia · 1 center
  • Monash Children s Hospital ( Site 1603) — Clayton
Belgium · 1 center
  • UZ Antwerpen ( Site 0601) — Edegem
Czechia · 1 center
  • Fakultni nemocnice v Motole ( Site 0700) — Prague
Finland · 1 center
  • New Childrens Hospital ( Site 0800) — Helsinki
Germany · 1 center
  • Universitaetsklinikum Freiburg ( Site 2203) — Freiburg im Breisgau
Netherlands · 1 center
  • Amsterdam UMC, locatie AMC ( Site 1000) — Amsterdam
New Zealand · 1 center
  • New Zealand Clinical Research (Christchurch) ( Site 1700) — Christchurch
Singapore · 1 center
  • National University Hospital-Paediatrics ( Site 1800) — Singapore

Identifiers

NCT: NCT07058077 · 0616-029 · U1111-1314-5796 · 2024-519068-42-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗