CLEAR Path 1: A Phase 2 Clinical Study in Children With HeFH Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing

Sponsor

Esperion Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05694260

Collaborator

(none)

Enrollment

Location

Arms

16.4

Anticipated Duration (Months)

3.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Hypercholesterolemia	Drug: Bempedoic acid	Phase 2

Detailed Description

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

Study Design

Study Type:

Interventional

Anticipated Enrollment :

54 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Jan 12, 2023

Anticipated Primary Completion Date :

Mar 24, 2024

Anticipated Study Completion Date :

May 24, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1 Patients at 16 to <30 kg body weight at screening receiving once daily 60 mg bempedoic acid for 8 weeks followed by 90 mg bempedoic acid for 8 weeks.	Drug: Bempedoic acid Once daily oral dosing with oral tablets or oral suspension. Other Names: ETC-1002
Experimental: Cohort 2 Patients 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 Once daily oral dosing with oral tablets or oral suspension. Other Names: ETC-1002
Experimental: Cohort 3 Patients at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks.	Drug: Bempedoic acid Once daily oral dosing with oral tablets or oral suspension. Other Names: ETC-1002

Arm

Intervention/Treatment

Experimental: Cohort 1

Patients at 16 to <30 kg body weight at screening receiving once daily 60 mg bempedoic acid for 8 weeks followed by 90 mg bempedoic acid for 8 weeks.

Drug: Bempedoic acid

Once daily oral dosing with oral tablets or oral suspension.

Other Names:

ETC-1002

Experimental: Cohort 2

Patients 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

Once daily oral dosing with oral tablets or oral suspension.

Other Names:

ETC-1002

Experimental: Cohort 3

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

Drug: Bempedoic acid

Once daily oral dosing with oral tablets or oral suspension.

Other Names:

ETC-1002

Outcome Measures

Primary Outcome Measures

Observed pharmacokinetics - trough plasma concentration of ETC-1002 [8 weeks of steady-state dosing]
observed trough plasma concentration of ETC-1002
Model-based pharmacokinetics - AUC, ss [8 weeks of steady-state dosing]
Model-based area under the plasma concentration-time curve (AUC,ss)
Model-based pharmacokinetics - Cavg, ss [8 weeks of steady-state dosing]
Model-based average plasma concentration (Cavg,ss)
Model-based pharmacokinetics - Cmax, ss [8 weeks of steady-state dosing]
Model-based maximum plasma concentration (Cmax,ss)

Secondary Outcome Measures

Exposure/LDL-C response relationship [8 weeks of steady-state dosing]
ETC-1002 dose and exposure/LDL-C-lowering response relationship
Percent change from baseline in LDL-C [8 weeks of steady-state dosing]
Percent change from baseline to Week 8 in LDL-C
Adverse Events [8 weeks of steady-state dosing]
Observed adverse events

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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