A Phase 2 Clinical Study in Children With HeFH Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing

Hypercholesterolemia Clinical Trial

— CLEAR Path 1  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05694260
• Other study ID #: 1002-041
• Status: Recruiting
• Phase: Phase 2
• Start date: January 12, 2023
• Est. completion date: May 24, 2024

Study information

• Verified date: May 2023
• Source: Esperion Therapeutics, Inc.
• Contact: Esperion Medical Information
• Phone: 833-377-7633
• Email: medinfo[@]esperion.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Multiple-dose study to measure PK, PD and safety of bempedoic acid in pediatric patients 6 to 17 years of age with HeFH.

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: May 24, 2024
• Est. primary completion date: March 24, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 17 Years

Eligibility

Inclusion Criteria:

Written informed consent and assent (where applicable) Aged 6-17 years old Diagnosis of HeFH (heterozygous familial hypercholesterolemia) Treatment with approved stable lipid modifying therapies Fasting LDL-C greater than or equal to 130 mg/dL (3.4 mmol/L)

Exclusion Criteria:

Diagnosis of HoFH (homozygous familial hypercholesterolemia) or compound HeFH Fasting Triglyceride greater than or equal to 400 mg/dL (4.5 mmol/L) Type 1 or Type 2 diabetes or newly diagnosed impaired glucose tolerance Women/girls who are pregnant or breastfeeding

Related Conditions & MeSH terms

• Hypercholesterolemia

Intervention

Drug:
• Bempedoic acid
Once daily oral dosing with oral tablets or oral suspension.

Locations

Country	Name	City	State
Canada	Ecogene-21	Chicoutimi	Quebec
Canada	University of Alberta Hospital - Stollery Children's Hospital	Edmonton	Alberta
Canada	McMaster University Medical Center	Hamilton	Ontario
Canada	The Hospital for Sick Children (SickKids)	Toronto	Ontario
Denmark	Rigshospitalet	Copenhagen
Germany	Kinder- und Jugendkrankenhaus AUF DER BULT	Hannover
Netherlands	Amsterdam UMC - Locatie AMC	Amsterdam
Netherlands	Erasmus MC	Rotterdam
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 Abente y Lago	La Coruña
Spain	Hospital Universitario Ramon y Cajal	Madrid
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Smidt Heart Institute at Cedars-Sinai	Los Angeles	California
United States	Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research.	Saint Louis	Missouri
United States	University of Utah and Primary Children's Hospital	Salt Lake City	Utah
United States	Continental Clinical Solutions	Towson	Maryland
United States	Wake Forest University Health Sciences	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Esperion Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Denmark,  Germany,  Netherlands,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Observed pharmacokinetics - trough plasma concentration of ETC-1002	observed trough plasma concentration of ETC-1002	8 weeks of steady-state dosing
Primary	Model-based pharmacokinetics - AUC, ss	Model-based area under the plasma concentration-time curve (AUC,ss)	8 weeks of steady-state dosing
Primary	Model-based pharmacokinetics - Cavg, ss	Model-based average plasma concentration (Cavg,ss)	8 weeks of steady-state dosing
Primary	Model-based pharmacokinetics - Cmax, ss	Model-based maximum plasma concentration (Cmax,ss)	8 weeks of steady-state dosing
Secondary	Exposure/LDL-C response relationship	ETC-1002 dose and exposure/LDL-C-lowering response relationship	8 weeks of steady-state dosing
Secondary	Percent change from baseline in LDL-C	Percent change from baseline to Week 8 in LDL-C	8 weeks of steady-state dosing
Secondary	Adverse Events	Observed adverse events	8 weeks of steady-state dosing