Esta página foi traduzida automaticamente e a precisão da tradução não é garantida. Por favor, consulte o versão em inglês para um texto fonte.

Um estudo clínico de fase 2 em crianças com HFHe de 6 a 17 anos tratadas uma vez ao dia com dosagem oral de ácido bempedoico (CLEAR Path 1)

15 de julho de 2026 atualizado por: Esperion Therapeutics, Inc.

Um estudo aberto para avaliar a farmacocinética, farmacodinâmica e segurança do ácido bempedóico em pacientes pediátricos (6 a 17 anos de idade) com hipercolesterolemia familiar heterozigótica

Estudo de dose múltipla para medir PK, DP e segurança do ácido bempedóico em pacientes pediátricos de 6 a 17 anos de idade com HFHe.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Descrição detalhada

A seleção da dose com base no peso corporal será determinada para uso no desenvolvimento clínico pediátrico

Tipo de estudo

Intervencional

Inscrição (Real)

31

Estágio

  • Fase 2

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Alemanha
      • Frankfurt am Main, Alemanha
        • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
      • Hanover, Alemanha
        • 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
  • Espanha
      • Barcelona, Espanha, 8950
        • Hospital Sant Joan de Déu
      • Barcelona, Espanha, 8208
        • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
      • Cadiz, Espanha, 11407
        • Hospital Universitario de Jerez de la Frontera
      • Córdoba, Espanha, 14004
        • Hospital Universitario Reina Sofia
      • Madrid, Espanha, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Espanha
        • Hospital Universitario 12 de Octubre
    • Galicia
      • A Coruña, Galicia, Espanha, 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
  • Holanda
      • Amsterdam, Holanda, 1105 AZ
        • Amsterdam UMC - Locatie AMC
      • Rotterdam, Holanda, 3015 G
        • Erasmus MC

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

6 anos a 17 anos (Filho)

Aceita Voluntários Saudáveis

Não

Descrição

Critério de inclusão:

Consentimento informado e consentimento por escrito (quando aplicável) De 6 a 17 anos de idade Diagnóstico de HFHe (hipercolesterolemia familiar heterozigótica) Tratamento com terapias modificadoras de lipídios estáveis ​​aprovadas LDL-C em jejum maior ou igual a 130 mg/dL (3,4 mmol/L)

Critério de exclusão:

Diagnóstico de HoFH (hipercolesterolemia familiar homozigótica) ou composto HeFH Triglicérides em jejum maior ou igual a 400 mg/dL (4,5 mmol/L) Diabetes tipo 1 ou tipo 2 ou intolerância à glicose recém-diagnosticada Mulheres/meninas grávidas ou amamentando

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Não randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Coorte 1
Os participantes de 16 a <30 kg (kg) de peso corporal na triagem recebendo uma vez por dia 60 miligramas (mg) ácido bem -pedóico por 8 semanas, seguidos por 90 mg de ácido bem -pedóico por 8 semanas.
Medicamento: Ácido bem -pedóico
Uma vez diariamente dosagem oral com comprimidos orais.
Outros nomes:
  • ETC-1002
Experimental: Coorte 2
Os participantes de 30 a 60 kg de peso corporal na triagem recebem uma vez por dia120 mg de ácido bem -devastado por 8 semanas, seguidos por 150 mg de ácido bempedóico por 8 semanas.
Medicamento: Ácido bem -pedóico
Uma vez diariamente dosagem oral com comprimidos orais.
Outros nomes:
  • ETC-1002
Experimental: Coorte 3
Participantes com maior que 60 kg de peso corporal na triagem recebendo uma vez ao dia 180 mg ácido bem -dedo por 8 semanas.
Medicamento: Ácido bem -pedóico
Uma vez diariamente dosagem oral com comprimidos orais.
Outros nomes:
  • ETC-1002

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Observed Trough Plasma Concentration of ETC-1002 Following 8 Weeks of Steady State Dosing of Bempedoic Acid
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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 resultados secundários

Medida de resultado
Descrição da medida
Prazo
Observed Trough Plasma Concentration of ESP15228
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Esperion Therapeutics, Inc.

Investigadores

  • Diretor de estudo: Jeffrey C Hanselman, MS, Esperion Therapeutics, Inc.

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

12 de janeiro de 2023

Conclusão Primária (Real)

4 de junho de 2025

Conclusão do estudo (Real)

4 de junho de 2025

Datas de inscrição no estudo

Enviado pela primeira vez

12 de janeiro de 2023

Enviado pela primeira vez que atendeu aos critérios de CQ

12 de janeiro de 2023

Primeira postagem (Real)

23 de janeiro de 2023

Atualizações de registro de estudo

Última Atualização Postada (Real)

17 de julho de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

15 de julho de 2026

Última verificação

1 de julho de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • 1002-041

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

Descrição do plano IPD

O Investigador deve garantir que a confidencialidade do paciente seja mantida. Os nomes e identidades de todos os pacientes da pesquisa serão mantidos em absoluto sigilo e não aparecerão em eCRFs ou outros registros fornecidos ou retidos pelo Patrocinador (ou pessoa designada). Se o nome de um paciente aparecer em qualquer documento, ele deve ser redigido e substituído pelo identificador do paciente antes que uma cópia do documento seja fornecida ao Patrocinador (ou pessoa designada). O TCLE deve incluir declarações apropriadas explicando que os dados do paciente serão confidenciais e as ações que serão tomadas para garantir a confidencialidade do paciente.

Quaisquer outros requisitos de confidencialidade especificados pelo site, IRB ou IEC, ou regulamentos nacionais ou locais serão cumpridos e detalhados adequadamente no ICF.

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

produto fabricado e exportado dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

Se inscrever