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Could a daily pill help kids with inherited high cholesterol?

NCT ID NCT05694260

Disease control Sponsor: Esperion Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 17, 2026 · Last updated Jul 17, 2026

Summary

This study tests bempedoic acid, a cholesterol-lowering tablet, in children aged 6 to 17 with heterozygous familial hypercholesterolemia (HeFH), an inherited condition causing very high cholesterol. Researchers measure how the drug moves through the body and how well it lowers cholesterol over 8 weeks. The goal is to find a safe and effective dose for this age group.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
bempedoic acid, a daily tablet that lowers cholesterol
What this could lead to
If successful, this could provide a new treatment option for children with inherited high cholesterol who cannot take or need more than statins.
What could go wrong
This is a small, early-phase study focused on drug levels and safety, not yet proving long-term benefit. Side effects or lack of effectiveness may emerge in larger trials.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

31 people

The number who actually took part.

Started

Jan 2023

Finished

Jun 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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). Other protocol defined inclusion and exclusion criteria.

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Conditions

The condition(s) this trial relates to.

familial hypercholesterolemia Hypercholesterolemia hyperlipidemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hypercholesterolemia

Contacts and locations

Locations

  • Amsterdam UMC - Locatie AMC

    Amsterdam, 1105 AZ, Netherlands

  • Cardiology Care for Children

    Lancaster, Pennsylvania, 17601, United States

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

    Barcelona, 8208, Spain

  • Ecogene-21

    Chicoutimi, Quebec, G7H 5H6, Canada

  • Erasmus MC

    Rotterdam, 3015 G, Netherlands

  • Excel Medical Clinical Trials, LLC

    Boca Raton, Florida, 33434, United States

  • Hospital Abente y Lago

    A Coruña, Galicia, 15001, Spain

  • Hospital Sant Joan de Deu

    Barcelona, 8950, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, Spain

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Reina Sofia

    Córdoba, 14004, Spain

  • Hospital Universitario de Jerez de la Frontera

    Cadiz, 11407, Spain

  • Kinder- und Jugendkrankenhaus AUF DER BULT

    Hanover, Germany

  • McMaster University Medical Center

    Hamilton, Ontario, L8N 3Z5, Canada

  • Providere Research Inc

    West Covina, California, 91790, United States

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

    Frankfurt am Main, Germany

  • University of Alberta Hospital - Stollery Children's Hospital

    Edmonton, Alberta, T6G 2B7, Canada

  • University of Utah and Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • Viborg Regional Hospital

    Viborg, Denmark

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157, United States

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

    St Louis, Missouri, 63110, United States

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