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)

NCT ID: NCT05694260

Last Updated: 2025-08-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 31 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-12
• Study Completion Date: 2025-06-04

Brief Summary

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

Detailed Description

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

Conditions

Hypercholesterolemia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1

Participants at 16 to \<30 kilograms (kg) body weight at screening receiving once daily 60 milligrams (mg) bempedoic acid for 8 weeks followed by 90 mg bempedoic acid for 8 weeks.

Group Type: EXPERIMENTAL

Bempedoic acid

Intervention Type: DRUG

Once daily oral dosing with oral tablets.

Cohort 2

Participants at 30 to 60 kg body weight at screening receiving once daily120 mg bempedoic acid for 8 weeks followed by 150 mg bempedoic acid for 8 weeks.

Group Type: EXPERIMENTAL

Bempedoic acid

Intervention Type: DRUG

Once daily oral dosing with oral tablets.

Cohort 3

Participants at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks.

Group Type: EXPERIMENTAL

Bempedoic acid

Intervention Type: DRUG

Once daily oral dosing with oral tablets.

Interventions

Bempedoic acid

Once daily oral dosing with oral tablets.

Intervention Type: DRUG

Other Intervention Names

ETC-1002

Eligibility Criteria

Inclusion Criteria

• Participant's parent(s)/guardian(s) must be willing to provide written informed consent and the participant must provide informed assent before any study-specific procedures 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 study medication at Treatment Visit T1 per Make Early Diagnosis to Prevent Early Deaths project (MEDPED) criteria by meeting at least one of the following clinical criteria:

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]) or TC >270 mg/dL (7.0 mmol/L), with no first- second- or third-degree relative with documented 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 with documented 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 relative with 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 an optimal dose of statin with or without other LMT(s), at stable dose for at least 4 weeks prior to Treatment Visit T1 (6 weeks for fibrates; however, gemfibrozil is not allowed in participants taking a statin as per coadministration instructions defined in the statin label). Participants must remain on that stable dose throughout the duration of the trial. Optimal dose of statin will be determined by the investigator using their medical judgment and available sources, including the participant's self-reported history of LMT. A participant's optimal dose of statin is defined as meeting one of the following criteria:

1. the highest approved dose of statin prescribed for the age of the participant based on regional practice or local guidelines; or

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

3. Participant/parent and investigator attestation to the participant's unwillingness to attempt and/or physician advice to not attempt additional statin therapy will be recorded.

Exclusion Criteria

• Participant has a diagnosis of homozygous familial hypercholesterolemia (HoFH) or compound HeFH;
• Participant has a fasting triglyceride (TG) level ≥400 mg/dL (4.5 mmol/L);
• Participant has uncontrolled hypothyroidism, including a value for thyroid-stimulating hormone (TSH) < lower limit of normal (LLN) or >1.5 × the upper limit of normal (ULN);
• Participant has liver disease or dysfunction, including:

1. positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis C virus 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 TB value is ≥1.2 × ULN, a reflex indirect (unconjugated) bilirubin will be obtained and, if consistent with Gilbert's disease or if the participant has a history of Gilbert's disease, the participant may be enrolled in the study.

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Esperion Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Cedars-Sinai Medical Center

Los Angeles, California, United States

Providere Research Inc

West Covina, California, United States

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, United States

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

St Louis, Missouri, United States

Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Cardiology Care for Children

Lancaster, Pennsylvania, United States

University of Utah and Primary Children's Hospital

Salt Lake City, Utah, United States

University of Alberta Hospital - Stollery Children's Hospital

Edmonton, Alberta, Canada

McMaster University Medical Center

Hamilton, Ontario, Canada

Ecogene-21

Chicoutimi, Quebec, Canada

Rigshospitalet

Copenhagen, , Denmark

Viborg Regional Hospital

Viborg, , Denmark

Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

Frankfurt am Main, , Germany

Kinder- und Jugendkrankenhaus AUF DER BULT

Hanover, , Germany

Amsterdam UMC - Locatie AMC

Amsterdam, , Netherlands

Erasmus MC

Rotterdam, , Netherlands

Hospital Abente y Lago

A Coruña, Galicia, Spain

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

Barcelona, , Spain

Hospital Sant Joan de Deu

Barcelona, , Spain

Hospital Universitario de Jerez de la Frontera

Cadiz, , Spain

Hospital Universitario Reina Sofia

Córdoba, , Spain

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Hospital Universitario 12 de Octubre

Madrid, , Spain

Countries

United States; Canada; Denmark; Germany; Netherlands; Spain

Other Identifiers

1002-041

Identifier Type: -

Identifier Source: org_study_id