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En klinisk fase 2 studie hos barn med HeFH i alderen 6 til 17 år behandlet én gang daglig med oral dosering av bempedosyre (CLEAR Path 1)

15. juli 2026 oppdatert av: Esperion Therapeutics, Inc.

En åpen studie for å evaluere farmakokinetikken, farmakodynamikken og sikkerheten til bempedosyre hos pediatriske pasienter (6 til 17 år) med heterozygot familiær hyperkolesterolemi

Flerdosestudie for å måle PK, PD og sikkerhet av bempedosyre hos pediatriske pasienter i alderen 6 til 17 år med HeFH.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Dosevalg basert på kroppsvekt vil bli bestemt for bruk i pediatrisk klinisk utvikling

Studietype

Intervensjonell

Registrering (Faktiske)

31

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

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
  • Forente stater
    • California
      • Los Angeles, California, Forente stater, 90048
        • Cedars-Sinai Medical Center
      • West Covina, California, Forente stater, 91790
        • Providere Research Inc
    • Florida
      • Boca Raton, Florida, Forente stater, 33434
        • Excel Medical Clinical Trials, LLC
    • Missouri
      • St Louis, Missouri, Forente stater, 63110
        • Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
    • North Carolina
      • Winston-Salem, North Carolina, Forente stater, 27157
        • Wake Forest University Health Sciences
    • Ohio
      • Cincinnati, Ohio, Forente stater, 45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Lancaster, Pennsylvania, Forente stater, 17601
        • Cardiology Care for Children
    • Utah
      • Salt Lake City, Utah, Forente stater, 84113
        • University of Utah and Primary Children's Hospital
  • Nederland
      • Amsterdam, Nederland, 1105 AZ
        • Amsterdam UMC - Locatie AMC
      • Rotterdam, Nederland, 3015 G
        • Erasmus MC
  • Spania
      • Barcelona, Spania, 8950
        • Hospital Sant Joan de Déu
      • Barcelona, Spania, 8208
        • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
      • Cadiz, Spania, 11407
        • Hospital Universitario de Jerez de la Frontera
      • Córdoba, Spania, 14004
        • Hospital Universitario Reina Sofia
      • Madrid, Spania, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spania
        • Hospital Universitario 12 de Octubre
    • Galicia
      • A Coruña, Galicia, Spania, 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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

6 år til 17 år (Barn)

Tar imot friske frivillige

Nei

Beskrivelse

Inklusjonskriterier:

Skriftlig informert samtykke og samtykke (der det er aktuelt) I alderen 6-17 år Diagnose av HeFH (heterozygot familiær hyperkolesterolemi) Behandling med godkjente stabile lipidmodifiserende terapier Fastende LDL-C større enn eller lik 130 mg/dL (3,4 mmol/L)

Ekskluderingskriterier:

Diagnose av HoFH (homozygot familiær hyperkolesterolemi) eller sammensatt HeFH fastende triglyserid større enn eller lik 400 mg/dL (4,5 mmol/L) Type 1 eller Type 2 diabetes eller nylig diagnostisert nedsatt glukosetoleranse Kvinner/jenter som er gravide eller ammer

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Kohort 1
Deltakere ved 16 til <30 kilo (kg) kroppsvekt ved screening mottar en gang daglig 60 milligram (mg) bempedoinsyre i 8 uker etterfulgt av 90 mg bempedoinsyre i 8 uker.
Legemiddel: Bempedoinsyre
En gang daglig oral dosering med orale tabletter.
Andre navn:
  • ETC-1002
Eksperimentell: Kohort 2
Deltakere ved 30 til 60 kg kroppsvekt ved screening som mottok en gang daglig 120 mg bempedoinsyre i 8 uker etterfulgt av 150 mg bempedoinsyre i 8 uker.
Legemiddel: Bempedoinsyre
En gang daglig oral dosering med orale tabletter.
Andre navn:
  • ETC-1002
Eksperimentell: Kohort 3
Deltakere med større enn 60 kg kroppsvekt ved screening som mottar en gang daglig 180 mg bempedoinsyre i 8 uker.
Legemiddel: Bempedoinsyre
En gang daglig oral dosering med orale tabletter.
Andre navn:
  • ETC-1002

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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
Tiltaksbeskrivelse
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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Esperion Therapeutics, Inc.

Etterforskere

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

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

12. januar 2023

Primær fullføring (Faktiske)

4. juni 2025

Studiet fullført (Faktiske)

4. juni 2025

Datoer for studieregistrering

Først innsendt

12. januar 2023

Først innsendt som oppfylte QC-kriteriene

12. januar 2023

Først lagt ut (Faktiske)

23. januar 2023

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

17. juli 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

15. juli 2026

Sist bekreftet

1. juli 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 1002-041

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

IPD-planbeskrivelse

Etterforskeren skal sørge for at pasientens konfidensialitet opprettholdes. Navnene og identitetene til alle forskningspasienter vil bli holdt strengt konfidensielt og vil ikke vises på eCRF-er eller andre poster som er gitt til eller oppbevart av sponsoren (eller den utpekte). Hvis en pasients navn vises på et dokument, må det redigeres og erstattes med pasientidentifikatoren før en kopi av dokumentet leveres til sponsoren (eller utpekt). ICF må inkludere passende erklæringer som forklarer at pasientdata vil være konfidensielle og handlingene som vil bli iverksatt for å sikre pasientens konfidensialitet.

Eventuelle andre konfidensialitetskrav spesifisert av nettstedet, IRB eller IEC, eller nasjonale eller lokale forskrifter, vil bli overholdt og detaljert på riktig måte i ICF.

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere