HypercholesterolemiaPhase 2NCT04784442

A Dose-finding Trial of ETC-1002(Bempedoic Acid) in Patients With Hypercholesterolemia

A Phase 2 study testing 180mg of ETC-1002(bempedoic acid), 120mg of ETC-1002(bempedoic acid) and 60mg of ETC-1002(bempedoic acid) in people with Hypercholesterolemia, run by Otsuka Pharmaceutical Co., Ltd. at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to assess the low-density lipoprotein cholesterol (LDL-C)-lowering efficacy and safety of ETC-1002(bempedoic acid) 60 mg, 120 mg and 180 mg versus placebo added to ongoing stable statin therapy or other lipid-modifying therapies in Japanese patients with hypercholesterolemia treated for 12… Read more about this study

Who is it for?: Ages 20 to 74 years
Conditions the study is for: Hypercholesterolemia
What is being tested?: 180mg of ETC-1002(bempedoic acid), 120mg of ETC-1002(bempedoic acid) and 60mg of ETC-1002(bempedoic acid)
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 188 participants
Where: 1 siteJapan
Study dates: 24 Mar 2021to 17 May 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 20 to 74 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients who have obtained informed consent to all of the observation/examination/evaluation items specified in the protocol
Patients must be on stable statin therapy defined as atorvastatin, pitavastatin, rosuvastatin, pravastatin, simvastatin, or fluvastatin daily[and other lipid-modifying therapies(LMTs) if needed] at least 4 weeks(6 weeks for fibrates) prior to screening and above LDL-C control target. Or Patients for statin intolerant must be on stable LMT(s) at least 4 weeks prior to screening and above LDL-C control target. Statin intolerance defined as an inability to tolerate 1 or more statins due to an adverse safety effect that started or increased during statin therapy and resolved or improved when statin therapy was discontinued or decreased. Patients on the lowest or under the dosage of the approved dose of statin or unable to tolerate any statin at any dose were eligible. Patients could continue taking the lowest or under the dosage of the approved dose of statin therapy or taking other LMTs throughout the study provided that it was stable and well tolerated.
Fasting mean TG level < 400 mg/dL from measurements at screening
Other protocol specific inclusion criteria may apply

Who cannot join

Women who are pregnant or breastfeeding or who have a positive pregnancy test (urine) result at screening or baseline visits
Sexually active male subjects or sexually active female subjects of childbearing potential who do not agree to practice 2 different methods of birth control or to remain abstinent during the trial and for 30 days after final IMP administration test (urine) result at screening or baseline visits
Patients with homozygous familial hypercholesterolemia (HoFH)
Patients with a history or current symptoms of any of the following clinically significant cardiovascular diseases within 3 months prior to screening or before baseline visit
Myocardial infarction, severe or unstable angina pectoris, coronary angioplasty, coronary artery bypass graft, stroke, transient ischemic attack, symptomatic carotid artery stenosis, symptomatic peripheral arterial disease, or decompensated heart failure
Abdominal aortic aneurysm

Show all 18Show fewer

Unexplained syncope or long-QT syndrome, family history of long-QT syndrome, or risk factors for Torsade de Pointes, such as persistent hypokalemia or second- or third-degree atrioventricular block (except when controlled by medication, etc)
Uncontrolled hypertension, defined as follows:
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 Hgb of <10.0 g/dL at screening
Patients with type 1 diabetes or uncontrolled type 2 diabetes with hemoglobin A1c (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) and/or hepatitis C antibodies at screening
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) of ≥3 × ULN and/or total bilirubin of ≥2 × ULN
Patients with creatine kinase (CK) elevation( >3 × ULN) at screening
Patients with renal dysfunction or nephritic syndrome or a history of nephritis and with estimated glomerular filtration rate (eGFR) of ≤30 mL/min/1.73m2 at screening
Other protocol specific inclusion criteria may apply

Trial locations and contacts

1 site in 1 country.

Japan1 site

Tokyo-Eki Center-Building Clinic
Chuo-ku,Tokyo

Other trials in Japan

About this study

Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial to Evaluate the Efficacy and Safety of ETC-1002 in Patients With Hypercholesterolemia

Conditions: Hypercholesterolemia
Being tested: 180mg of ETC-1002(bempedoic acid), 120mg of ETC-1002(bempedoic acid), 60mg of ETC-1002(bempedoic acid), Placebo
Medical subject headings: Hypercholesterolemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

180mg of ETC-1002(bempedoic acid)Drug
180mg, tablet, once daily, for 12 weeks
120mg of ETC-1002(bempedoic acid)Drug
120mg, tablet, once daily, for 12 weeks
60mg of ETC-1002(bempedoic acid)Drug
60mg, tablet, once daily, for 12 weeks
PlaceboDrug
placebo, tablet, once daily, for 12 weeks

Study groups in NCT04784442
Study group	Role	What they receive
ETC-1002 180mg	Experimental	Not described Receives: Drug: 180mg of ETC-1002(bempedoic acid)
ETC-1002 120mg	Experimental	Not described Receives: Drug: 120mg of ETC-1002(bempedoic acid)
ETC-1002 60mg	Experimental	Not described Receives: Drug: 60mg of ETC-1002(bempedoic acid)
Placebo	Placebo comparator	Not described Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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. When LDL-C at Week 12 was missing, the missing value was imputed using the last observation carried forward from the start of the IMP administration to 2 days after the final IMP administration.
Measured: Baseline, week12

Secondary outcomes (9)

Percent Change in HDL Cholesterol 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.
Measured: Baseline, week12
Percent Change in Non-HDL Cholesterol 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.
Measured: Baseline, week12
Percent Change in Total Cholesterol 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.
Measured: Baseline, week12
Percent Change in Triglycerides 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.
Measured: Baseline, week12
Percent Change in Apolipoprotein B 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.
Measured: Baseline, week12
Percent Change in High Sensitivity C Reactive Protein 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.
Measured: Baseline, week12
Percent Change in Hemoglobin A1c 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.
Measured: Baseline, week12
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 12
The proportion of subjects whose LDL-C value achieves the lipid management goal at Week 12.
Measured: Baseline, week12
Proportion of Subjects Whose LDL-C Value Achieve < 70 mg/dL at Week 12
The proportion of subjects whose LDL-C value achieves <70 mg/dL at Week 12.
Measured: Baseline, week12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Mar 202117 May 2022

First posted5 Mar 2021
Study start24 Mar 2021
Primary completion18 Apr 2022
Study completion17 May 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
More trials run by Otsuka Pharmaceutical Co., Ltd.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 188 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04784442
Sponsor's protocol ID: 346-102-00001
Other IDs: jRCT2031200445
Status last verified: May 2024
First submitted: 2 Mar 2021
Last updated: 10 May 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Yes: : Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Study Evaluating LDL-C Change and Adherence to Inclisiran Lipid-lowering Therapy in ASCVD
Primary Hypercholesterolemia, Mixed Dyslipidemia
Active, not recruitingPhase 3
A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
Primary Prevention of Atherosclerotic Cardiovascular Disease
RecruitingObservational study
Causes and Natural History of Dyslipidemias
Hypercholesterolemia, Atherosclerosis
Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia

All Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 10 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial to Evaluate the Efficacy and Safety of ETC-1002 in Patients With Hypercholesterolemia
ClinicalTrials.gov ID: NCT04784442
Acronym / sponsor ID: 346-102-00001
Conditions: Hypercholesterolemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 188 participants (actual)
Lead sponsor: Otsuka Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
Study start: 24 Mar 2021 (actual)
Primary completion: 18 Apr 2022 (actual)
Study completion: 17 May 2022 (actual)
First posted: 5 Mar 2021 (actual)
Results: Posted 4 Mar 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 4

Arm	Type	Receives
ETC-1002 180mg	Experimental	Drug: 180mg of ETC-1002(bempedoic acid)
ETC-1002 120mg	Experimental	Drug: 120mg of ETC-1002(bempedoic acid)
ETC-1002 60mg	Experimental	Drug: 60mg of ETC-1002(bempedoic acid)
Placebo	Placebo comparator	Drug: Placebo

Who may qualify

Ages: 20 to 74 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Patients who have obtained informed consent to all of the observation/examination/evaluation items specified in the protocol
Patients must be on stable statin therapy defined as atorvastatin, pitavastatin, rosuvastatin, pravastatin, simvastatin, or fluvastatin daily[and other lipid-modifying therapies(LMTs) if needed] at least 4 weeks(6 weeks for fibrates) prior to screening and above LDL-C control target. Or Patients for statin intolerant must be on stable LMT(s) at least 4 weeks prior to screening and above LDL-C control target. Statin intolerance defined as an inability to tolerate 1 or more statins due to an adverse safety effect that started or increased during statin therapy and resolved or improved when statin therapy was discontinued or decreased. Patients on the lowest or under the dosage of the approved dose of statin or unable to tolerate any statin at any dose were eligible. Patients could continue taking the lowest or under the dosage of the approved dose of statin therapy or taking other LMTs throughout the study provided that it was stable and well tolerated.
Fasting mean TG level < 400 mg/dL from measurements at screening
Other protocol specific inclusion criteria may apply

Key exclusion criteria (18 listed)

Women who are pregnant or breastfeeding or who have a positive pregnancy test (urine) result at screening or baseline visits
Sexually active male subjects or sexually active female subjects of childbearing potential who do not agree to practice 2 different methods of birth control or to remain abstinent during the trial and for 30 days after final IMP administration test (urine) result at screening or baseline visits
Patients with homozygous familial hypercholesterolemia (HoFH)
Patients with a history or current symptoms of any of the following clinically significant cardiovascular diseases within 3 months prior to screening or before baseline visit
Uncontrolled hypertension, defined as follows:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
180mg of ETC-1002(bempedoic acid)180mg, tablet, once daily, for 12 weeks	Drug	ETC-1002 180mg
120mg of ETC-1002(bempedoic acid)120mg, tablet, once daily, for 12 weeks	Drug	ETC-1002 120mg
60mg of ETC-1002(bempedoic acid)60mg, tablet, once daily, for 12 weeks	Drug	ETC-1002 60mg
Placeboplacebo, tablet, once daily, for 12 weeks	Drug	Placebo

Primary outcome

Percent Change in LDL-C From Baseline to Week 12
Time frame: 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. When LDL-C at Week 12 was missing, the missing value was imputed using the last observation carried forward from the start of the IMP administration to 2 days after the final IMP administration.

9 secondary outcomes

Percent Change in HDL Cholesterol From Baseline to Week 12
Time frame: Baseline, week12
Percent Change in Non-HDL Cholesterol From Baseline to Week 12
Time frame: Baseline, week12
Percent Change in Total Cholesterol From Baseline to Week 12
Time frame: Baseline, week12
Percent Change in Triglycerides From Baseline to Week 12
Time frame: Baseline, week12
Percent Change in Apolipoprotein B From Baseline to Week 12
Time frame: Baseline, week12
Percent Change in High Sensitivity C Reactive Protein From Baseline to Week 12
Time frame: Baseline, week12
Percent Change in Hemoglobin A1c From Baseline to Week 12
Time frame: Baseline, week12
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 12
Time frame: Baseline, week12
Proportion of Subjects Whose LDL-C Value Achieve < 70 mg/dL at Week 12
Time frame: Baseline, week12

Results and safety

Results posted to the registry on 4 Mar 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ETC-1002 60 mg	47	44
ETC-1002 120 mg	46	46
ETC-1002 180 mg	48	46
Placebo	47	46

Primary outcome: Percent Change in LDL-C From Baseline to Week 12

Time frame: Baseline, week12. Least squares mean (Standard Error). Unit: Percent Change

Measure	ETC-1002 60 mg n = 45	ETC-1002 120 mg n = 46	ETC-1002 180 mg n = 48	Placebo n = 47
Value	-10.59 (2.153)	-21.85 (2.156)	-21.26 (2.095)	-1.92 (2.117)

ETC-1002 180 mg vs Placebo: Least squares mean difference: -19.35 (95% CI -24.81 to -13.88), p <0.001, ANCOVA
ETC-1002 120 mg vs Placebo: Least squares mean difference: -19.93 (95% CI -25.45 to -14.41), p <0.001, ANCOVA
ETC-1002 60 mg vs Placebo: Least squares mean difference: -8.67 (95% CI -14.22 to -3.12), p = 0.002, ANCOVA

Population analysed: The FAS included 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 (up to 2 days after final IMP administration) are observed.

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication.. Other adverse events reported at 4% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ETC-1002 60 mg	0 of 47 (0%)	23 of 47 (48.9%)	0 of 47
ETC-1002 120 mg	0 of 46 (0%)	17 of 46 (37%)	0 of 46
ETC-1002 180 mg	1 of 47 (2.1%)	21 of 48 (43.8%)	0 of 47
Placebo	1 of 48 (2.1%)	9 of 47 (19.1%)	0 of 48

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. : Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal.

Related trials for Hypercholesterolemia

Active, not recruitingObservational study
Study Evaluating LDL-C Change and Adherence to Inclisiran Lipid-lowering Therapy in ASCVD
Primary Hypercholesterolemia, Mixed Dyslipidemia
Active, not recruitingPhase 3
A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
Primary Prevention of Atherosclerotic Cardiovascular Disease
RecruitingObservational study
Causes and Natural History of Dyslipidemias
Hypercholesterolemia, Atherosclerosis
Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Takehisa Matsumaru study director

Otsuka Pharmaceutical Co., Ltd.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change in LDL-C From Baseline to Week 12 (time frame: Baseline, week12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04784442.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2024.