COMPLETED

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)

Conditions

Hypercholesterolemia Pediatric Heterozygous familial hypercholesterolemia Low-density lipoprotein cholesterol (LDL-cholesterol)Bempedoic acid ETC-1002 Adenosine triphosphate citrate lyase

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic Acid in Pediatric Patients (6 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia

Quick Facts

Study Start:2023-01-12

Study Completion:2025-06-04

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:COMPLETED

Study ID

NCT05694260

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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: * 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.	* 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).

Inclusion Criteria

Exclusion 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:
* 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.

* 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).

Contacts and Locations

Study Locations (Sites)

Cedars-Sinai Medical Center

Los Angeles, California, 90048

United States

Providere Research Inc

West Covina, California, 91790

United States

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, 33434

United States

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

Saint Louis, Missouri, 63110

United States

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

United States

Cardiology Care for Children

Lancaster, Pennsylvania, 17601

United States

University of Utah and Primary Children's Hospital

Salt Lake City, Utah, 84113

United States

Collaborators and Investigators

Sponsor: Esperion Therapeutics, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-01-12

Study Completion Date2025-06-04

Study Record Updates

Study Start Date2023-01-12

Study Completion Date2025-06-04

Terms related to this study

Keywords Provided by Researchers

Pediatric
Heterozygous familial hypercholesterolemia
Low-density lipoprotein cholesterol (LDL-cholesterol)
Bempedoic acid
ETC-1002
Adenosine triphosphate citrate lyase

Additional Relevant MeSH Terms

Hypercholesterolemia