ReclutandoNCT07058077Registro de ClinicalTrials.gov

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

Heterozygous Familial Hypercholesterolemia (HeFH)

Pregúntale a WCR sobre este estudio Registro oficial ↗

LO ESENCIAL

De qué trata este estudio

El registro indica que este estudio recluta participantes. La disponibilidad puede variar por centro.

Tipo de estudio: De intervención
Fase: Fase 2 / Fase 3
Rango de edad: 6 años - 17 años
Sexo admitido: Todos los sexos
Voluntarios sanos: No aceptados
Participantes: 153 Estimado

Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.

Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.

Acerca de la investigación

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.

¿Quién podría participar?

La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.

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

Entiende la evaluación inicial

Tratamiento, grupos y participación

El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.

Asignación: Asignación al azar
Enmascaramiento: Enmascaramiento cuádruple

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

Part B: Enlicitide Decanoate

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

Drug: Enlicitide Decanoate

Part B: Placebo

Participants receive placebo orally QD for up to 24 weeks.

Drug: Placebo

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 DRUG

Enlicitide decanoate taken by mouth

Placebo DRUG

Placebo tablet matched to enlicitide decanoate taken by mouth

Prepara tus preguntas

Qué evalúan los investigadores

Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.

Medidas principales

Part A: Maximum Plasma Concentration (Cmax) of Enlicitide

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

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

Part A: Area Under the Concentration-Time Curve from 0 to 24 Hours (AUC0-24) of Enlicitide

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

Blood samples will be collected to determine the AUC0-24 of enlicitide.

Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C)

Plazo: Baseline and Week 24

Blood samples will be collected to determine the percent change from baseline in LDL-C.

Number of Participants Who Experience an Adverse Event (AE)

Plazo: Up to approximately 188 weeks

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.

Number of Participants Who Discontinue Study Treatment Due to an AE

Plazo: Up to approximately 180 weeks

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.