A Confirmatory Trial of ETC-1002 in Patients With Hyper-LDL Cholesterolemia

A Placebo-controlled, Randomized, Multicenter, Double-blind, Parallel-group Trial to Confirm the Superiority of ETC-1002 in Patients With Hyper-LDL Cholesterolemia

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05683340

Enrollment

Registered

2023-01-13

Start date

2023-02-13

Completion date

2024-03-12

Last updated

2025-03-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hyper-low-density Lipoprotein (LDL) Cholesterolemia

Brief summary

The purpose of this study is to confirm the superiority of ETC-1002 after 12 weeks of administration at 180 mg/day to placebo in patients with hyper-LDL cholesterolemia who have inadequate control of low-density lipoprotein cholesterol (LDL C).

Interventions

DRUG180mg of ETC-1002(bempedoic acid)

180mg, tablet, once daily, for 12 weeks

DRUGPlacebo

placebo, tablet, once daily, for 12 weeks

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 85 Years

Healthy volunteers

Inclusion criteria

* Patients with inadequate response to statins or statin intolerance as defined below \[Inadequate response to statins\] Patients with hyper-LDL cholesterolemia who have been taking statins\[and other lipid-modifying therapies(LMTs) if needed\] and cannot achieve the lipid management goals of LDL-C \[Statin intolerance\] Patients with hyper-LDL cholesterolemia for whom safety problems have occurred while taking at least one type of statin, and who experienced resolution of the problems after discontinuation or dose reduction, and who cannot achieve the lipid management goals of LDL-C. Patients must be on the lowest or under the dosage of the approved dose of statin and/or on stable LMT(s). * Patients with fasting TG levels of \<400 mg/dL at screening * Other protocol specific inclusion criteria may apply

Exclusion criteria

* Females who are pregnant or breast-feeding or who have a positive pregnancy test (urine) result at screening or baseline visits * Patients with homozygous familial hypercholesterolemia (HoFH) * Patients who currently have or who have had within the past 3 months prior to screening any cardiovascular diseases, or those who have developed any cardiovascular diseases during the screening or before baseline visit * Uncontrolled hypertension, defined as sitting systolic blood pressure after resting 5 minutes of ≥160 mmHg or diastolic blood pressure of ≥100 mmHg at screening * Patients with uncontrolled and serious hematologic or coagulation disorders or with hemoglobin of \<10.0 g/dL at screening * Patients with uncontrolled diabetes with HbA1c of ≥9% at screening * Patients with uncontrolled hypothyroidism with thyroid-stimulating hormone (TSH) of \>1.5 × ULN at screening * Patients with liver disease or dysfunction, including: * Positive serology for hepatitis B surface antigen (HBsAg) or a positive hepatitis C virus (HCV) antibody test at screening * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) of ≥3 × ULN or total bilirubin of ≥2 × ULN at screening * Patients with creatine kinase (CK) of \>3 × ULN at screening * Patients with a history or current renal dysfunction, nephritic syndrome, or nephritis, and with estimated glomerular filtration rate (eGFR) of ≤30 mL/min/1.73 m2 at screening * Other protocol specific

Design outcomes

Primary

Measure	Time frame	Description
Percent Change in LDL-C From Baseline to Week 12	Baseline, week12	Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100. Baseline was defined as the mean of the values from Week -1 and Day 1.

Secondary

Measure	Time frame	Description
Percent Change in Total Cholesterol From Baseline to Week 12	Baseline, week12	Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100.
Percent Change in Apolipoprotein B From Baseline to Week 12	Baseline, week12	Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100.
Percent Change in Non-HDL Cholesterol From Baseline to Week 12	Baseline, week12	Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100.
Percent Change in Hemoglobin A1c From Baseline to Week 12	Baseline, week12	Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100.
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 12	Baseline, week12	The proportion of subjects whose LDL-C value achieves the lipid management goal at Week 12.
Percent Change in High Sensitivity C Reactive Protein From Baseline to Week 12	Baseline, week12	Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100.

Countries

Japan

Participant flow

Participants by arm

Arm	Count
ETC-1002 180 mg ETC-1002 180 mg tablet once daily for 12 weeks.	48
Placebo Placebo tablet once daily for 12 weeks.	48
Total	96

Withdrawals & dropouts

Period	Reason	FG000	FG001
Overall Study	Adverse Event	1	1
Overall Study	Protocol Violation	1	0
Overall Study	Withdrawal by Subject	0	2

Baseline characteristics

Characteristic	ETC-1002 180 mg	Total	Placebo
Age, Categorical <=18 years	0 Participants	0 Participants	0 Participants
Age, Categorical >=65 years	28 Participants	51 Participants	23 Participants
Age, Categorical Between 18 and 65 years	20 Participants	45 Participants	25 Participants
Age, Continuous	64.6 years STANDARD_DEVIATION 13.02	64.2 years STANDARD_DEVIATION 12.93	63.9 years STANDARD_DEVIATION 12.98
Race/Ethnicity, Customized Japanese	48 Participants	96 Participants	48 Participants
Region of Enrollment Japan	48 participants	96 participants	48 participants
Sex: Female, Male Female	28 Participants	51 Participants	23 Participants
Sex: Female, Male Male	20 Participants	45 Participants	25 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	0 / 48	0 / 48
other Total, other adverse events	13 / 48	6 / 48
serious Total, serious adverse events	0 / 48	0 / 48

Outcome results

Primary

Percent Change in LDL-C From Baseline to Week 12

Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100. Baseline was defined as the mean of the values from Week -1 and Day 1.

Time frame: Baseline, week12

Population: The full analysis set (FAS) will include all subjects who receive at least one dose of IMP during the treatment period and for whom LDL-C values at baseline and at least one post-dose are observed.

Arm	Measure	Value (LEAST_SQUARES_MEAN)	Dispersion
ETC-1002 180 mg	Percent Change in LDL-C From Baseline to Week 12	-25.25 Percent Change	Standard Error 1.864
Placebo	Percent Change in LDL-C From Baseline to Week 12	-3.46 Percent Change	Standard Error 1.901

p-value: 0.00195% CI: [-26.71, -16.85]MMRM

Secondary