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

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

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

Official Title
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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

Conditions
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Hypercholesterolemia

Intervention / Treatment
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* Drug: Bempedoic acid


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1<br>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<br>* Once daily oral dosing with oral tablets or oral suspension.<br>* Other names: ETC-1002;|
| Experimental: Cohort 2<br>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<br>* Once daily oral dosing with oral tablets or oral suspension.<br>* Other names: ETC-1002;|
| Experimental: Cohort 3<br>Patients at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks. | Drug: Bempedoic acid<br>* Once daily oral dosing with oral tablets or oral suspension.<br>* Other names: ETC-1002;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Observed pharmacokinetics - trough plasma concentration of ETC-1002 | observed trough plasma concentration of ETC-1002 | 8 weeks of steady-state dosing | 
| Model-based pharmacokinetics - AUC, ss | Model-based area under the plasma concentration-time curve (AUC,ss) | 8 weeks of steady-state dosing | 
| Model-based pharmacokinetics - Cavg, ss | Model-based average plasma concentration (Cavg,ss) | 8 weeks of steady-state dosing | 
| Model-based pharmacokinetics - Cmax, ss | Model-based maximum plasma concentration (Cmax,ss) | 8 weeks of steady-state dosing | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Exposure/LDL-C response relationship | ETC-1002 dose and exposure/LDL-C-lowering response relationship | 8 weeks of steady-state dosing | 
| Percent change from baseline in LDL-C | Percent change from baseline to Week 8 in LDL-C | 8 weeks of steady-state dosing | 
| Adverse Events | Observed adverse events | 8 weeks of steady-state dosing | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pediatric, HeFH, LDL-cholesterol, ETC-1002, Adenosine triphosphate citrate lyase