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Et fase 2 klinisk studie i børn med HeFH i alderen 6 til 17 år behandlet én gang dagligt med oral dosering af bempedosyre (CLEAR Path 1)

15. juli 2026 opdateret af: Esperion Therapeutics, Inc.

En åben-label undersøgelse til evaluering af farmakokinetikken, farmakodynamikken og sikkerheden af ​​bempedosyre hos pædiatriske patienter (6 til 17 år) med heterozygot familiær hyperkolesterolæmi

Multipel-dosis undersøgelse til måling af PK, PD og sikkerhed af bempedoinsyre hos pædiatriske patienter i alderen 6 til 17 år med HeFH.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

Dosisvalg baseret på kropsvægt vil blive bestemt til brug i pædiatrisk klinisk udvikling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

31

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

6 år til 17 år (Barn)

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

Skriftligt informeret samtykke og samtykke (hvor relevant) I alderen 6-17 år Diagnose af HeFH (heterozygot familiær hyperkolesterolæmi) Behandling med godkendte stabile lipidmodificerende terapier Fastende LDL-C større end eller lig med 130 mg/dL (3,4 mmol/L)

Ekskluderingskriterier:

Diagnose af HoFH (homozygot familiær hyperkolesterolæmi) eller sammensat HeFH fastende triglycerid større end eller lig med 400 mg/dL (4,5 mmol/L) Type 1 eller Type 2 diabetes eller nydiagnosticeret nedsat glukosetolerance Kvinder/piger, der er gravide eller ammer

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Kohort 1
Deltagere ved 16 til <30 kg (kg) kropsvægt ved screening, der modtager en gang dagligt 60 milligram (Mg) Bempedoesyre i 8 uger efterfulgt af 90 mg Bempedoesyre i 8 uger.
Medicin: Bempedoesyre
En gang daglig oral dosering med orale tabletter.
Andre navne:
  • ETC-1002
Eksperimentel: Kohort 2
Deltagerne ved 30 til 60 kg kropsvægt ved screening, der modtog en gang dagligt120 mg Bempedoinsyre i 8 uger efterfulgt af 150 mg Bempedoesyre i 8 uger.
Medicin: Bempedoesyre
En gang daglig oral dosering med orale tabletter.
Andre navne:
  • ETC-1002
Eksperimentel: Kohort 3
Deltagerne på mere end 60 kg kropsvægt ved screening, der modtog en gang dagligt 180 mg Bempedoinsyre i 8 uger.
Medicin: Bempedoesyre
En gang daglig oral dosering med orale tabletter.
Andre navne:
  • ETC-1002

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Observed Trough Plasma Concentration of ETC-1002 Following 8 Weeks of Steady State Dosing of Bempedoic Acid
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Observed Trough Plasma Concentration of ESP15228
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Esperion Therapeutics, Inc.

Efterforskere

  • Studieleder: Jeffrey C Hanselman, MS, Esperion Therapeutics, Inc.

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

12. januar 2023

Primær færdiggørelse (Faktiske)

4. juni 2025

Studieafslutning (Faktiske)

4. juni 2025

Datoer for studieregistrering

Først indsendt

12. januar 2023

Først indsendt, der opfyldte QC-kriterier

12. januar 2023

Først opslået (Faktiske)

23. januar 2023

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

17. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

15. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • 1002-041

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

IPD-planbeskrivelse

Investigator skal sikre, at patientens fortrolighed opretholdes. Navne og identiteter på alle forskningspatienter vil blive holdt strengt fortrolige og vil ikke blive vist på eCRF'er eller andre optegnelser, som er givet til eller opbevaret af sponsoren (eller den udpegede). Hvis en patients navn optræder på et dokument, skal det redigeres og erstattes med patientidentifikationen, før en kopi af dokumentet leveres til sponsoren (eller den udpegede). ICF skal inkludere passende erklæringer, der forklarer, at patientdata vil være fortrolige, og de handlinger, der vil blive truffet for at sikre patientens fortrolighed.

Eventuelle andre fortrolighedskrav specificeret af webstedet, IRB eller IEC eller nationale eller lokale regler vil blive overholdt og detaljeret i ICF.

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Abonner