A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)

Last updated: February 26, 2025
Sponsor: Esperion Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Familial Hypercholesterolemia

Hypercholesterolemia

Treatment

Bempedoic acid

Clinical Study ID

NCT05694260
1002-041
  • Ages 6-17
  • All Genders

Study Summary

Multiple-dose study to measure pharmacokinetics, pharmacodynamics and safety of bempedoic acid in pediatric participants 6 to 17 years of age with HeFH.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Participant's parent(s)/guardian(s) must be willing to provide written informedconsent and the participant must provide informed assent before any study-specificprocedures are performed;

  • Participant must be aged 6-17 years old and willing to swallow tablets;

  • Participant must weigh at least 16 kilograms (kg);

  • Participant must have a diagnosis of HeFH prior to receiving the first dose of studymedication at Treatment Visit T1 per Make Early Diagnosis to Prevent Early Deathsproject (MEDPED) criteria by meeting at least one of the following clinicalcriteria:

  1. Documented diagnosis of HeFH determined by positive genetic testing; or

  2. Documented LDL-C or TC meeting one or more of the following criteria: i. LDL-C >200 milligrams per deciliter (mg/dL) (5.2 millimole per liter [mmol/L]) orTC >270 mg/dL (7.0 mmol/L), with no first- second- or third-degree relative withdocumented FH diagnosis (general population); or ii. LDL-C >155 mg/dL (4.0 mmol/L)or TC >220 mg/dL (5.7 mmol/L), and also having a first-degree relative withdocumented familial hypercholesterolemia (FH) diagnosis; or iii. LDL-C >165 mg/dL (4.3 mmol/L) or TC >230 mg/dL (5.9 mmol/L), and also having a second-degree relativewith documented FH diagnosis; or iv. LDL-C >170 mg/dL (4.4 mmol/L) or TC >240 mg/dL (6.2 mmol/L), and also having a third-degree relative with documented FH diagnosis

  • Current treatment with approved stable lipid-modifying therapy (LMT), including anoptimal dose of statin with or without other LMT(s), at stable dose for at least 4weeks prior to Treatment Visit T1 (6 weeks for fibrates; however, gemfibrozil is notallowed in participants taking a statin as per coadministration instructions definedin the statin label). Participants must remain on that stable dose throughout theduration of the trial. Optimal dose of statin will be determined by the investigatorusing their medical judgment and available sources, including the participant'sself-reported history of LMT. A participant's optimal dose of statin is defined asmeeting one of the following criteria:
  1. the highest approved dose of statin prescribed for the age of the participantbased on regional practice or local guidelines; or

  2. less than the highest approved dose of statin, including no statin, prescribedfor the age of the participant based on regional practice or local guidelines (including no statin) if: i. the participant has previously taken 2 or morestatin therapies at any dose and not able to tolerate or unresponsive due totheir mutations (null); or ii. the participant has previously taken 1 or morestatin therapies at any dose and is unwilling to attempt another statin at anydose or advised by a physician to not attempt another statin at any dose.

  3. Participant/parent and investigator attestation to the participant'sunwillingness to attempt and/or physician advice to not attempt additionalstatin therapy will be recorded.

Exclusion

Exclusion Criteria:

  • Participant has a diagnosis of homozygous familial hypercholesterolemia (HoFH) orcompound HeFH;

  • Participant has a fasting triglyceride (TG) level ≥400 mg/dL (4.5 mmol/L);

  • Participant has uncontrolled hypothyroidism, including a value forthyroid-stimulating hormone (TSH) < lower limit of normal (LLN) or >1.5 × the upperlimit of normal (ULN);

  • Participant has liver disease or dysfunction, including:

  1. positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis Cvirus antibodies (HCV-AB), or

  2. serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value ≥2 × ULN and/or serum total bilirubin (TB) value ≥2 × ULN. If the serum TBvalue is ≥1.2 × ULN, a reflex indirect (unconjugated) bilirubin will beobtained and, if consistent with Gilbert's disease or if the participant has ahistory of Gilbert's disease, the participant may be enrolled in the study.

  • Participant has renal dysfunction or glomerulonephritis, including an estimatedglomerular filtration rate (eGFR) <75 milliliters/minute/1.73 square meter (mL/min/1.73 m^2).

Other protocol defined inclusion and exclusion criteria.

Study Design

Total Participants: 54
Treatment Group(s): 1
Primary Treatment: Bempedoic acid
Phase: 2
Study Start date:
January 12, 2023
Estimated Completion Date:
August 31, 2025

Study Description

Dose-selection based on body weight will be determined for use in pediatric clinical development.

Connect with a study center

  • University of Alberta Hospital - Stollery Children's Hospital

    Edmonton, Alberta T6G 2B7
    Canada

    Active - Recruiting

  • McMaster University Medical Center

    Hamilton, Ontario L8N 3Z5
    Canada

    Active - Recruiting

  • The Hospital for Sick Children (SickKids)

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • Ecogene-21

    Chicoutimi, Quebec G7H 5H6
    Canada

    Active - Recruiting

  • Rigshospitalet

    Copenhagen, 2100
    Denmark

    Active - Recruiting

  • Viborg Regional Hospital

    Viborg,
    Denmark

    Active - Recruiting

  • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

    Frankfurt am Main,
    Germany

    Active - Recruiting

  • Kinder- und Jugendkrankenhaus AUF DER BULT

    Hannover,
    Germany

    Active - Recruiting

  • Amsterdam UMC - Locatie AMC

    Amsterdam, 1105 AZ
    Netherlands

    Active - Recruiting

  • Erasmus MC

    Rotterdam, 3015 G
    Netherlands

    Active - Recruiting

  • Hospital Abente y Lago

    La Coruña, Galicia 15001
    Spain

    Active - Recruiting

  • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

    Barcelona, 8208
    Spain

    Active - Recruiting

  • Hospital Sant Joan de Deu

    Barcelona, 8950
    Spain

    Active - Recruiting

  • Hospital Universitario de Jerez de la Frontera

    Cadiz, 11407
    Spain

    Active - Recruiting

  • Hospital Universitario Reina Sofia

    Córdoba, 14004
    Spain

    Active - Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid,
    Spain

    Active - Recruiting

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034
    Spain

    Active - Recruiting

  • Cedars-Sinai Medical Center

    Los Angeles, California 90048
    United States

    Active - Recruiting

  • Smidt Heart Institute at Cedars-Sinai

    Los Angeles, California 90048
    United States

    Active - Recruiting

  • Providere Research Inc

    West Covina, California 91790
    United States

    Active - Recruiting

  • Excel Medical Clinical Trials, LLC

    Boca Raton, Florida 33434
    United States

    Active - Recruiting

  • Continental Clinical Solutions

    Towson, Maryland 21204
    United States

    Site Not Available

  • Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research.

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina 27157
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • Cardiology Care for Children

    Lancaster, Pennsylvania 17601
    United States

    Active - Recruiting

  • University of Utah and Primary Children's Hospital

    Salt Lake City, Utah 84113
    United States

    Active - Recruiting

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