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

Официальное название

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

Обзор

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.

Вмешательства

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

Первичные конечные точки

• Part A: Maximum Plasma Concentration (Cmax) of Enlicitide [Срок оценки: 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 [Срок оценки: At designated timepoints (up to 24 hours postdose on day 14)]
• Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) [Срок оценки: Baseline and Week 24]
• Number of Participants Who Experience an Adverse Event (AE) [Срок оценки: Up to approximately 188 weeks]
• Number of Participants Who Discontinue Study Treatment Due to an AE [Срок оценки: Up to approximately 180 weeks]

Вторичные конечные точки (7)

• Part B: Percent Change from Baseline in Apolipoprotein B (ApoB) [Срок оценки: Baseline and week 24]
• Part B: Percent Change from Baseline in Non-High-Density Lipoprotein Cholesterol (non-HDL-C) [Срок оценки: Baseline and week 24]
• Part B: Percent Change from Baseline in Lipoprotein (a) (Lp(a)) [Срок оценки: Baseline and week 24]
• Part B: Percentage of Participants With LDL-C <130 mg/dL at Week 24 [Срок оценки: Week 24]
• Part B: Percentage of Participants With ≥50% LDL-C Reduction from Baseline at Week 24 [Срок оценки: Baseline and week 24]
• Part B: Percentage of Participants With LDL-C <100 mg/dL at Week 24 [Срок оценки: Week 24]
• Change in Carotid Intima-media Thickness (cIMT) [Срок оценки: Baseline and week 24]

Критерии участия

Критерии включения

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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 7 центров

• 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

Китай · 5 центров

• Beijing Anzhen Hospital. Capital Medical University ( Site 1917) — Пекин
• Guangdong Provincial People's Hospital ( Site 1915) — Гуанчжоу
• Children's Hospital of Fudan University ( Site 1906) — Шанхай
• Shanghai Children's Medical Center ( Site 1918) — Шанхай
• The Children's Hospital of Zhejiang University School of Medicine ( Site 1905) — Ханчжоу

Колумбия · 4 центра

• 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

Бразилия · 3 центра

• Universidade Federal Do Ceara ( Site 0201) — Fortaleza
• Instituto Dante Pazzanese de Cardiology ( Site 0206) — São Paulo
• Incor - Instituto do Coracao ( Site 0200) — São Paulo

Мексика · 3 центра

• 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

Испания · 3 центра

• 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

Великобритания · 3 центра

• Birmingham Childrens Hospital ( Site 1501) — Birmingham
• Sheffield Childrens Hospital ( Site 1503) — Sheffield
• Southampton General Hospital ( Site 1502) — Southampton

Чили · 2 центра

• Explora Salud ( Site 0304) — Santiago
• Pontificia Universidad Catolica de Chile ( Site 0300) — Santiago

Австралия · 1 центр

• Monash Children s Hospital ( Site 1603) — Clayton

Бельгия · 1 центр

• UZ Antwerpen ( Site 0601) — Edegem

Чехия · 1 центр

• Fakultni nemocnice v Motole ( Site 0700) — Prague

Финляндия · 1 центр

• New Childrens Hospital ( Site 0800) — Helsinki

Германия · 1 центр

• Universitaetsklinikum Freiburg ( Site 2203) — Freiburg im Breisgau

Нидерланды · 1 центр

• Amsterdam UMC, locatie AMC ( Site 1000) — Amsterdam

Новая Зеландия · 1 центр

• New Zealand Clinical Research (Christchurch) ( Site 1700) — Christchurch

Сингапур · 1 центр

• National University Hospital-Paediatrics ( Site 1800) — Singapore

Идентификаторы

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

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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