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CompletedNCT04784442Updated May 10, 2024Results posted

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

A Phase 2 interventional study of 180mg of ETC-1002(bempedoic acid) and 120mg of ETC-1002(bempedoic acid) in Hypercholesterolemia, sponsored by Otsuka Pharmaceutical Co., Ltd.. Completed at 1 site in Japan. Open to participants aged 20 Years to 74 Years. Per ClinicalTrials.gov, last updated 2024-05-10.

Sponsored by Otsuka Pharmaceutical Co., Ltd. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
188
Allocation
Randomized
Ages
20 Years to 74 Years
Sex
All
01

Study 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 weeks.

02

Conditions studied

  • Hypercholesterolemia
03

In context

Hypercholesterolemia

1,238 studies on the registry are indexed under Hypercholesterolemia; 109 are open to participants now.

This study's enrollment of 188 is above the median of 99 across 991 interventional studies indexed under Hypercholesterolemia.

Browse Hypercholesterolemia studies →

Lead sponsor

Otsuka Pharmaceutical Co., Ltd. is the lead sponsor of 140 studies on the registry; 11 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
20 Years to 74 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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

Exclusion criteria

Exclusion Criteria:

  • 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
    • 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
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
188 participants (actual)

Study arms

  • Experimental
    ETC-1002 180mg

    Drug: 180mg of ETC-1002(bempedoic acid)

  • Experimental
    ETC-1002 120mg

    Drug: 120mg of ETC-1002(bempedoic acid)

  • Experimental
    ETC-1002 60mg

    Drug: 60mg of ETC-1002(bempedoic acid)

  • Placebo comparator
    Placebo

    Drug: Placebo

Interventions

  • Drug180mg of ETC-1002(bempedoic acid)

    180mg, tablet, once daily, for 12 weeks

  • Drug120mg of ETC-1002(bempedoic acid)

    120mg, tablet, once daily, for 12 weeks

  • Drug60mg of ETC-1002(bempedoic acid)

    60mg, tablet, once daily, for 12 weeks

  • DrugPlacebo

    placebo, tablet, once daily, for 12 weeks

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline, week12

Secondary outcomes

  1. 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.

    Time frame: Baseline, week12

  2. 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.

    Time frame: Baseline, week12

  3. 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.

    Time frame: Baseline, week12

  4. 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.

    Time frame: Baseline, week12

  5. 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.

    Time frame: Baseline, week12

  6. 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.

    Time frame: Baseline, week12

  7. 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.

    Time frame: Baseline, week12

  8. 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.

    Time frame: Baseline, week12

  9. 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.

    Time frame: Baseline, week12

07

Results

Posted Mar 4, 2024

Participant flow

Participant flow — Overall Study
MilestoneETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Started47464847
Completed44464646
Not completed3021
Withdrew: Adverse event1021
Withdrew: Protocol violation1000
Withdrew: Non-compliance with study drug1000

Outcome measures

PrimaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in LDL-C From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in LDL-C From Baseline to Week 12-10.59 ± 2.153-21.85 ± 2.156-21.26 ± 2.095-1.92 ± 2.117
Statistical analysis
  • ETC-1002 180 mg vs Placebo · ANCOVA · p = <0.001 · Least squares mean difference: -19.35 · 95% CI -24.81 to -13.88The least squares mean difference was calculated as the value for the ETC-1002 180 mg arm minus the value for the placebo arm.
  • ETC-1002 120 mg vs Placebo · ANCOVA · p = <0.001 · Least squares mean difference: -19.93 · 95% CI -25.45 to -14.41The least squares mean difference was calculated as the value for the ETC-1002 120 mg arm minus the value for the placebo arm.
  • ETC-1002 60 mg vs Placebo · ANCOVA · p = 0.002 · Least squares mean difference: -8.67 · 95% CI -14.22 to -3.12
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in HDL Cholesterol From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in HDL Cholesterol From Baseline to Week 120.16 ± 2.208-0.65 ± 2.190-7.29 ± 2.1393.08 ± 2.155
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in Non-HDL Cholesterol From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in Non-HDL Cholesterol From Baseline to Week 12-8.86 ± 2.019-18.29 ± 2.021-16.01 ± 1.956-1.62 ± 1.988
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in Total Cholesterol From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in Total Cholesterol From Baseline to Week 12-6.42 ± 1.665-13.61 ± 1.650-13.56 ± 1.599-0.55 ± 1.621
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in Triglycerides From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in Triglycerides From Baseline to Week 12-9.09 ± 6.286-5.62 ± 6.2909.17 ± 6.151-0.08 ± 6.219
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in Apolipoprotein B From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in Apolipoprotein B From Baseline to Week 12-8.18 ± 1.715-14.59 ± 1.709-12.47 ± 1.704-2.13 ± 1.704
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in High Sensitivity C Reactive Protein From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in High Sensitivity C Reactive Protein From Baseline to Week 12150.54 ± 62.4941.02 ± 62.418-34.58 ± 62.7450.98 ± 61.513
SecondaryPercent 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.

Time frame:
Baseline, week12
Reported as:
Least squares mean · Percent Change
Percent Change in Hemoglobin A1c From Baseline to Week 12
Percent ChangeETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Percent Change in Hemoglobin A1c From Baseline to Week 120.11 ± 0.591-0.40 ± 0.596-0.21 ± 0.592-0.05 ± 0.578
SecondaryProportion 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.

Time frame:
Baseline, week12
Reported as:
Number · participants
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 12
participantsETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 121526281
SecondaryProportion 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.

Time frame:
Baseline, week12
Reported as:
Count of participants · Participants
Proportion of Subjects Whose LDL-C Value Achieve < 70 mg/dL at Week 12
ParticipantsETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Proportion of Subjects Whose LDL-C Value Achieve < 70 mg/dL at Week 120100

Adverse events

Collected over 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.. Non-serious events are listed at a 4% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
ETC-1002 60 mg0/47 (0%)0/47 (0%)23/47 (48.9%)
ETC-1002 120 mg0/46 (0%)0/46 (0%)17/46 (37%)
ETC-1002 180 mg0/47 (0%)1/47 (2.1%)21/48 (43.8%)
Placebo0/48 (0%)1/48 (2.1%)9/47 (19.1%)
Most frequent serious events
Most frequent serious events
EventETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
Calculus urinaryRenal and urinary disorders0/470/461/470/48
Dental cystGastrointestinal disorders0/470/460/471/48
Most frequent other events
Showing 10 of 25
Most frequent other events
EventETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlacebo
HeadacheNervous system disorders0/475/462/480/47
PyrexiaGeneral disorders5/474/461/482/47
Hepatic function abnormalHepatobiliary disorders2/470/465/480/47
Abdominal pain upperGastrointestinal disorders4/471/460/480/47
MalaiseGeneral disorders1/473/463/480/47
Vaccination site painGeneral disorders1/473/460/481/47
Blood uric acid increasedInvestigations2/473/461/480/47
DiarrhoeaGastrointestinal disorders3/471/461/481/47
ArthralgiaMusculoskeletal and connective tissue disorders3/472/462/480/47
MyalgiaMusculoskeletal and connective tissue disorders1/471/463/483/47

Baseline characteristics

For the full analysis set (FAS), a total of 186 subjects (45 for the 60 mg group, 46 for the 120 mg group, and 48 for the 180 mg group were included in the ETC-1002 group, and 47 subjects were included in the placebo group).

Age, Categorical
Age, Categorical(Participants)ETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlaceboTotal
<=18 years00000
Between 18 and 65 years2827232098
>=65 years1719252788
Age, Continuous
Age, Continuous(years)ETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlaceboTotal
Mean60.9 ± 9.2660.4 ± 8.9460.4 ± 11.9863.0 ± 10.6561.2 ± 10.28
Sex: Female, Male
Sex: Female, Male(Participants)ETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlaceboTotal
Female1616161260
Male29303235126
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)ETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlaceboTotal
Asian45464847186
Region of Enrollment
Region of Enrollment(participants)ETC-1002 60 mgETC-1002 120 mgETC-1002 180 mgPlaceboTotal
Japan45464847186
08

Study locations

1 site
  • Tokyo-Eki Center-Building Clinic
    Chuo-ku,Tokyo, Japan
09

References and documents

Study documents

  • Study protocol · Nov 19, 2021
  • Statistical analysis plan · Jun 20, 2022

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: 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.

Supporting information: Study protocol, Sap

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 10, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04784442
Lead sponsor
Otsuka Pharmaceutical Co., Ltd.
Responsible party
Sponsor
First posted
Mar 5, 2021
Start date
Mar 24, 2021
Primary completion
Apr 18, 2022
Completion
May 17, 2022
Results posted
Mar 4, 2024
Last update
May 10, 2024

Study contacts

Takehisa Matsumaru
study director · Otsuka Pharmaceutical Co., Ltd.

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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