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)

NCT05694260 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 1002-041
• Study Type: interventional
• Target: 31
• Locations: 6 countries
• Sites: 24

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.

Conditions

Hypercholesterolemia

Keywords

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

Outcome Measures

Primary Outcomes (4)

• Observed Trough Plasma Concentration of ETC-1002 Following 8 Weeks of Steady State Dosing of Bempedoic Acid

  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

  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

  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

  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

Secondary Outcomes (15)

• Observed Trough Plasma Concentration of ESP15228

  Week 8 pre-dose

• Observed Plasma Concentration at 4 Hours (C4hr) of ETC-1002

  Day 1: 4 hours post-dose

• Observed C4hr of ESP15228

  Day 1: 4 hours post-dose

• Simulated Percent Change From Baseline in Exposure/ Low-Density Lipoprotein-Cholesterol (LDL-C)

  Baseline and Week 12

• Observed Percent Change From Baseline in LDL-C

  Baseline and 8 Weeks post-treatment

Study Arms (3)

Cohort 1

EXPERIMENTAL

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.

Drug: Bempedoic acid

Cohort 2

EXPERIMENTAL

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.

Drug: Bempedoic acid

Cohort 3

EXPERIMENTAL

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

Drug: Bempedoic acid

Interventions

Bempedoic acid DRUG

Once daily oral dosing with oral tablets.

Also known as: ETC-1002

Cohort 1; Cohort 2; Cohort 3

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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:
• Documented diagnosis of HeFH determined by positive genetic testing; or
• 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:
• the highest approved dose of statin prescribed for the age of the participant based on regional practice or local guidelines; or
• 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.
• 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.

You may not qualify if:

• 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:
• positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis C virus antibodies (HCV-AB), or
• 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).

Sponsors & Collaborators

• Esperion Therapeutics, Inc.: lead

Study Sites (24)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Providere Research Inc

West Covina, California, 91790, United States

Location

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, 33434, United States

Location

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

St Louis, Missouri, 63110, United States

Location

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Cardiology Care for Children

Lancaster, Pennsylvania, 17601, United States

Location

University of Utah and Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

University of Alberta Hospital - Stollery Children's Hospital

Edmonton, Alberta, T6G 2B7, Canada

Location

McMaster University Medical Center

Hamilton, Ontario, L8N 3Z5, Canada

Location

Ecogene-21

Chicoutimi, Quebec, G7H 5H6, Canada

Location

Rigshospitalet

Copenhagen, 2100, Denmark

Location

Viborg Regional Hospital

Viborg, Denmark

Location

Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

Frankfurt am Main, Germany

Location

Kinder- und Jugendkrankenhaus AUF DER BULT

Hanover, Germany

Location

Amsterdam UMC - Locatie AMC

Amsterdam, 1105 AZ, Netherlands

Location

Erasmus MC

Rotterdam, 3015 G, Netherlands

Location

Hospital Abente y Lago

A Coruña, Galicia, 15001, Spain

Location

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

Barcelona, 8208, Spain

Location

Hospital Sant Joan de Deu

Barcelona, 8950, Spain

Location

Hospital Universitario de Jerez de la Frontera

Cadiz, 11407, Spain

Location

Hospital Universitario Reina Sofia

Córdoba, 14004, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, Spain

Location

MeSH Terms

Conditions

Hypercholesterolemia

Interventions

8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Medical Director
• Organization: Esperion Therapeutics, Inc.

medinfo@esperion.com

1-833-377-7633

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 12, 2023
• First Posted: January 23, 2023
• Study Start: January 12, 2023
• Primary Completion: June 4, 2025
• Study Completion: June 4, 2025
• Last Updated: July 17, 2026
• Results First Posted: July 17, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

DK (2); NL (2); US (8); CA (3); DE (2); ES (7)