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

Status: Completed
Location: See all (24) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 17
Healthy Volunteers: f
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• 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.

Locations
United States
California
Cedars-Sinai Medical Center
Los Angeles
Providere Research Inc
West Covina
Florida
Excel Medical Clinical Trials, LLC
Boca Raton
Missouri
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
St Louis
North Carolina
Wake Forest University Health Sciences
Winston-salem
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Pennsylvania
Cardiology Care for Children
Lancaster
Utah
University of Utah and Primary Children's Hospital
Salt Lake City
Other Locations
Canada
Ecogene-21
Chicoutimi
University of Alberta Hospital - Stollery Children's Hospital
Edmonton
McMaster University Medical Center
Hamilton
Denmark
Rigshospitalet
Copenhagen
Viborg Regional Hospital
Viborg
Germany
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
Frankfurt Am Main
Kinder- und Jugendkrankenhaus AUF DER BULT
Hanover
Netherlands
Amsterdam UMC - Locatie AMC
Amsterdam
Erasmus MC
Rotterdam
Spain
Hospital Abente y Lago
A Coruña
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Barcelona
Hospital Sant Joan de Deu
Barcelona
Hospital Universitario de Jerez de la Frontera
Cadiz
Hospital Universitario Reina Sofia
Córdoba
Hospital Universitario 12 de Octubre
Madrid
Hospital Universitario Ramon y Cajal
Madrid
Time Frame
Start Date: 2023-01-12
Completion Date: 2025-06-04
Participants
Target number of participants: 31
Treatments
Experimental: 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.
Experimental: 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.
Experimental: Cohort 3
Participants at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks.
Sponsors
Leads: Esperion Therapeutics, Inc.

This content was sourced from clinicaltrials.gov