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Un estudio clínico de fase 2 en niños con HFHe de 6 a 17 años tratados una vez al día con dosificación oral de ácido bempedoico (CLEAR Path 1)

15 de julio de 2026 actualizado por: Esperion Therapeutics, Inc.

Un estudio abierto para evaluar la farmacocinética, la farmacodinámica y la seguridad del ácido bempedoico en pacientes pediátricos (6 a 17 años de edad) con hipercolesterolemia familiar heterocigota

Estudio de dosis múltiples para medir PK, PD y seguridad del ácido bempedoico en pacientes pediátricos de 6 a 17 años con HeFH.

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Descripción detallada

Se determinará la selección de dosis basada en el peso corporal para su uso en el desarrollo clínico pediátrico

Tipo de estudio

Intervencionista

Inscripción (Actual)

31

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Alemania
      • Frankfurt am Main, Alemania
        • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
      • Hanover, Alemania
        • Kinder- und Jugendkrankenhaus AUF DER BULT
  • Canadá
    • Alberta
      • Edmonton, Alberta, Canadá, T6G 2B7
        • University of Alberta Hospital - Stollery Children's Hospital
    • Ontario
      • Hamilton, Ontario, Canadá, L8N 3Z5
        • McMaster University Medical Center
    • Quebec
      • Chicoutimi, Quebec, Canadá, G7H 5H6
        • Ecogene-21
  • Dinamarca
      • Copenhagen, Dinamarca, 2100
        • Rigshospitalet
      • Viborg, Dinamarca
        • Viborg Regional Hospital
  • España
      • Barcelona, España, 8950
        • Hospital Sant Joan de Déu
      • Barcelona, España, 8208
        • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
      • Cadiz, España, 11407
        • Hospital Universitario de Jerez de la Frontera
      • Córdoba, España, 14004
        • Hospital Universitario Reina Sofia
      • Madrid, España, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, España
        • Hospital Universitario 12 de Octubre
    • Galicia
      • A Coruña, Galicia, España, 15001
        • Hospital Abente y Lago
  • Estados Unidos
    • California
      • Los Angeles, California, Estados Unidos, 90048
        • Cedars-Sinai Medical Center
      • West Covina, California, Estados Unidos, 91790
        • Providere Research Inc
    • Florida
      • Boca Raton, Florida, Estados Unidos, 33434
        • Excel Medical Clinical Trials, LLC
    • Missouri
      • St Louis, Missouri, Estados Unidos, 63110
        • Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
    • North Carolina
      • Winston-Salem, North Carolina, Estados Unidos, 27157
        • Wake Forest University Health Sciences
    • Ohio
      • Cincinnati, Ohio, Estados Unidos, 45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Lancaster, Pennsylvania, Estados Unidos, 17601
        • Cardiology Care for Children
    • Utah
      • Salt Lake City, Utah, Estados Unidos, 84113
        • University of Utah and Primary Children's Hospital
  • Países Bajos
      • Amsterdam, Países Bajos, 1105 AZ
        • Amsterdam UMC - Locatie AMC
      • Rotterdam, Países Bajos, 3015 G
        • Erasmus MC

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

6 años a 17 años (Niño)

Acepta Voluntarios Saludables

No

Descripción

Criterios de inclusión:

Consentimiento informado por escrito y asentimiento (cuando corresponda) Edad de 6 a 17 años Diagnóstico de HeFH (hipercolesterolemia familiar heterocigota) Tratamiento con terapias modificadoras de lípidos estables aprobadas C-LDL en ayunas mayor o igual a 130 mg/dL (3,4 mmol/L)

Criterio de exclusión:

Diagnóstico de HoFH (hipercolesterolemia familiar homocigótica) o compuesto HeFH Triglicéridos en ayunas mayores o iguales a 400 mg/dL (4,5 mmol/L) Diabetes tipo 1 o tipo 2 o intolerancia a la glucosa recientemente diagnosticada Mujeres/niñas que están embarazadas o amamantando

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Cohorte 1
Participantes de 16 a <30 kilogramos (kg) de peso corporal en la detección que reciben una vez al día 60 miligramos (mg) ácido bemedoico durante 8 semanas seguido de 90 mg de ácido bemedoico durante 8 semanas.
Droga: Ácido bemedoico
Una vez dosis oral diaria con tabletas orales.
Otros nombres:
  • ETC-1002
Experimental: Cohorte 2
Participantes de 30 a 60 kg de peso corporal en la detección que reciben una vez al día120 mg de ácido bemedoico durante 8 semanas seguido de 150 mg de ácido bemedoico durante 8 semanas.
Droga: Ácido bemedoico
Una vez dosis oral diaria con tabletas orales.
Otros nombres:
  • ETC-1002
Experimental: Cohorte 3
Participantes de más de 60 kg de peso corporal en la detección que reciben una vez al día 180 mg de ácido bemedoico durante 8 semanas.
Droga: Ácido bemedoico
Una vez dosis oral diaria con tabletas orales.
Otros nombres:
  • ETC-1002

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Observed Trough Plasma Concentration of ETC-1002 Following 8 Weeks of Steady State Dosing of Bempedoic Acid
Periodo de tiempo: Week 8 pre-dose
Blood plasma samples were collected and analyzed to determine the plasma trough concentration of ETC-1002 following 8 weeks of steady-state dosing of bempedoic acid. The data presented here is for participants who received tablet formulation only.
Week 8 pre-dose
Model-based Steady State Area Under the Concentration-time Curve Over 24 Hours (AUC,24ss) of ETC-1002
Periodo de tiempo: 24 hours
Blood plasma samples were collected and analyzed to determine the AUC,24ss of ETC-1002 over 24 hours. The data presented here is for participants who received tablet formulation only.
24 hours
Model-based Steady State Average Plasma Concentration (Cavg,ss) of ETC-1002
Periodo de tiempo: Week 8, 24 hours post-dose at steady state
Blood plasma samples were collected and analyzed to determine the Cavg,ss of ETC-1002. Cavg was calculated by empirical Bayesian estimated pediatric exposure over 24 hours divided by 24 (AUC24hr.ss / 24). The data presented here is for participants who received tablet formulation only.
Week 8, 24 hours post-dose at steady state
Model-based Steady State Maximum Plasma Concentration (Cmax,ss) of ETC-1002
Periodo de tiempo: Day 1: 1-hour and 4 hours post-dose; Week 4 pre-dose; Week 8 pre-dose
Blood plasma samples were collected and analyzed to determine the Cmax,ss of ETC-1002. The data presented here is for participants who received tablet formulation only.
Day 1: 1-hour and 4 hours post-dose; Week 4 pre-dose; Week 8 pre-dose

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Observed Trough Plasma Concentration of ESP15228
Periodo de tiempo: Week 8 pre-dose
Blood plasma samples were collected and analyzed to determine the trough plasma concentration of active metabolite ESP15228 following 8 weeks of steady-state dosing of bempedoic acid. The data presented here is for participants who received tablet formulation only.
Week 8 pre-dose
Observed Plasma Concentration at 4 Hours (C4hr) of ETC-1002
Periodo de tiempo: Day 1: 4 hours post-dose
Blood plasma samples were collected and analyzed to determine the trough plasma concentration of ETC-1002 4 hours post-dose. The data presented here is for participants who received tablet formulation only.
Day 1: 4 hours post-dose
Observed C4hr of ESP15228
Periodo de tiempo: Day 1: 4 hours post-dose
Blood plasma samples were collected and analyzed to determine the trough plasma concentration of active metabolite ESP15228 4 hours post-dose on Day 1. The data presented here is for participants who received tablet formulation only.
Day 1: 4 hours post-dose
Simulated Percent Change From Baseline in Exposure/ Low-Density Lipoprotein-Cholesterol (LDL-C)
Periodo de tiempo: Baseline and Week 12
Blood samples were collected for the analysis of and exposure/LDL-C-lowering response relationship.
Baseline and Week 12
Observed Percent Change From Baseline in LDL-C
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of LDL-C levels. Baseline is defined as the last assessment measurements before the first dose of investigational medical product (IMP). Percent change from baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Absolute Change From Baseline in LDL-C (mg/dl)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of LDL-C levels. Baseline is defined as the last assessment measurements before the first dose of IMP. Change from baseline is defined as post- dose visit value minus baseline value. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of Non-HDL-C levels. Baseline is defined as the last assessment measurements before the first dose of IMP. Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Absolute Change From Baseline in Non-HDL-C (mg/dL)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of Non-HDL-C levels. Baseline is defined as the last assessment measurements before the first dose of IMP. Change from Baseline is defined as post- dose visit value minus Baseline value. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Percent Change From Baseline in Total Cholesterol (TC)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of total cholesterol levels. Baseline is defined as the last assessment measurements before the first dose of IMP. Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Absolute Change From Baseline in TC (mg/dL)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of TC levels. Baseline is defined as the last assessment measurements before the first dose of IMP. Change from Baseline is defined as post- dose visit value minus Baseline value. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of hsCRP. Baseline is defined as the last assessment measurements before the first dose of IMP. Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Observed Absolute Change From Baseline in hsCRP (mg/L)
Periodo de tiempo: Baseline and 8 Weeks post-treatment
Blood samples were collected for analysis of hsCRP levels. Baseline is defined as the last assessment measurements before the first dose of IMP. Change from Baseline is defined as post- dose visit value minus Baseline value. The treatment period for participants in cohort 1 (60 mg), cohort 2 (120 mg), and cohort 3 was weeks 1-8. The treatment period for participants in cohort 1 (90 mg) and cohort 2 (150 mg) was weeks 8-16.
Baseline and 8 Weeks post-treatment
Acceptability of Taste of Liquid Formulation Using a Dosing Acceptability Questionnaire
Periodo de tiempo: Days 1 and 2; Weeks 2, 4, 8, 8 (Phone), 10, 12, and 16
Participant acceptance is defined as the overall ability of the participant to use a medicine as intended. The acceptability of overall flavor of IMP was recorded using a dosing acceptability questionnaire, where participants were asked 'Yes' or 'No', whether the flavor of the medication was acceptable. At Week 8 (Phone) participants in cohorts 1 and 2 were contacted by phone to collect questionnaire information. The number of participants who have responded at each time point have been presented.
Days 1 and 2; Weeks 2, 4, 8, 8 (Phone), 10, 12, and 16
Acceptability of Ease of Swallowing Tablet Formulation Using a Dosing Acceptability Questionnaire
Periodo de tiempo: Days 1 and 2; Weeks 2, 4, 8, 8 (Phone), 10, 12, and 16
Participant acceptance is defined as the overall ability of the participant to use a medicine as intended. Acceptability of ease of swallowing was recorded using a dosing acceptability questionnaire where participants were asked if they were able to swallow the entire dose or not, by ticking 'yes' or 'no'. At Week 8 (Phone) participants in cohorts 1 and 2 were contacted by phone to collect questionnaire information. The number of participants who responded at each time point have been presented.
Days 1 and 2; Weeks 2, 4, 8, 8 (Phone), 10, 12, and 16
Number of Participants Reporting Serious Adverse Events (SAEs) and Non-SAEs
Periodo de tiempo: Up to Week 16
An AE is the development of an undesirable medical condition or the deterioration of a pre-existing medical condition following or during exposure to a pharmaceutical product, whether or not considered causally related to the product. A serious AE is an AE occurring during any study phase (i.e., baseline, treatment, washout, or follow-up), and at any dose of the study medication that fulfills one or more of the following: results in death, is immediately life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital abnormality or birth defect, is an important medical event that may jeopardize the participant or may require medical intervention to prevent one of the outcomes listed above.
Up to Week 16

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Esperion Therapeutics, Inc.

Investigadores

  • Director de estudio: Jeffrey C Hanselman, MS, Esperion Therapeutics, Inc.

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

12 de enero de 2023

Finalización primaria (Actual)

4 de junio de 2025

Finalización del estudio (Actual)

4 de junio de 2025

Fechas de registro del estudio

Enviado por primera vez

12 de enero de 2023

Primero enviado que cumplió con los criterios de control de calidad

12 de enero de 2023

Publicado por primera vez (Actual)

23 de enero de 2023

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

17 de julio de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

15 de julio de 2026

Última verificación

1 de julio de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 1002-041

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Descripción del plan IPD

El investigador debe asegurarse de que se mantenga la confidencialidad del paciente. Los nombres y las identidades de todos los pacientes de la investigación se mantendrán en estricta confidencialidad y no aparecerán en los eCRF u otros registros proporcionados o retenidos por el Patrocinador (o su designado). Si el nombre de un paciente aparece en cualquier documento, se debe redactar y reemplazar con el identificador del paciente antes de que se proporcione una copia del documento al Patrocinador (o designado). El ICF debe incluir declaraciones apropiadas que expliquen que los datos del paciente serán confidenciales y las acciones que se tomarán para garantizar la confidencialidad del paciente.

Cualquier otro requisito de confidencialidad especificado por el sitio, IRB o IEC, o las regulaciones nacionales o locales se cumplirá y se detallará adecuadamente en el ICF.

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

producto fabricado y exportado desde los EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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