NCT02666664

Brief Summary

The purpose of this study is to see if ETC-1002 (bempedoic acid) is safe and well-tolerated versus placebo in patients with high cardiovascular risk and elevated LDL cholesterol that is not adequately controlled by their current therapy.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2,230

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Jan 2016

Geographic Reach
6 countries

104 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 20, 2016

Completed
1 day until next milestone

Study Start

First participant enrolled

January 21, 2016

Completed
7 days until next milestone

First Posted

Study publicly available on registry

January 28, 2016

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 21, 2018

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

March 28, 2018

Completed
2.1 years until next milestone

Results Posted

Study results publicly available

April 20, 2020

Completed
Last Updated

May 11, 2020

Status Verified

April 1, 2020

Enrollment Period

2.1 years

First QC Date

January 20, 2016

Results QC Date

March 20, 2020

Last Update Submit

April 24, 2020

Conditions

Keywords

hyperlipidemiacholesterolheterozygous familial hypercholesterolemiaatherosclerotic cardiovascular diseaseASCVDHeFHLDL

Outcome Measures

Primary Outcomes (13)

  • Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

    TEAEs, defined as adverse events (AEs) that began or worsened in severity after the first dose of double-blind study drug and up to 30 days after receiving the last dose of double-blind study drug, were collected and reported.

    Up to approximately 52 weeks

  • Percentage of Participants With Adjudicated Major Adverse Cardiovascular Event

    TEAEs, defined as AEs that began or worsened in severity after the first dose of double-blind study drug and up to 30 days after receiving the last dose of double-blind study drug, were collected and reported. Cardiovascular events were considered as adverse events of special interest. Treatment-emergent = TE.

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Creatine Kinase Elevations

    TEAEs of special interest (AESIs) were predefined and monitored throughout the study. Creatine kinase elevations were assessed using the following preferred term: Blood creatine phosphokinase increased (system organ class: investigations).

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Hepatic Disorders

    Treatment-emergent AESIs were predefined and monitored throughout the study. TEAEs potentially related to hepatic events were assessed using the following preferred terms and laboratory abnormalities: aspartate aminotransferase (AST) increased, Alanine aminotransferase (ALT) increased, Hepatic enzyme increased, Blood bilirubin increased, liver function test (LFT) abnormal, LFT increased, hepatic enzyme abnormal, transaminases increased, potential Hy's Law cases (PHLC) \[AST and (\&)/or ALT \>3 x upper limit of normal (ULN) with concurrent total bilirubin \>2 x ULN\], AST and/or ALT \>3 x ULN, and total bilirubin \>2 x ULN (system organ class: investigations). AST and ALT values were repeated and confirmed. "Repeated and confirmed" was defined as a participant having the last on-study LFT \> x ULN, the last on-treatment LFT \> x ULN, or LFT \> x ULN followed by another LFT \> x ULN.

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Hypoglycemia

    Treatment-emergent AESIs were predefined and monitored throughout the study. Hypoglycemia was assessed using the following preferred terms: hypoglycaemia (system organ class: metabolism and nutrition disorders); blood glucose abnormal and blood glucose decreased (system organ class: investigations). The percentage of unique participants is reported in the "Overall hypoglycemia AESIs" category; a participant could have been represented in more than one of the individual hypoglycemia AESIs.

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Metabolic Acidosis

    Treatment-emergent AESIs were predefined and monitored throughout the study. Metabolic acidosis was assessed using the preferred term metabolic acidosis (system organ class: metabolism and nutrition disorders).

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Muscular Disorder

    Treatment-emergent AESIs were predefined and monitored throughout the study. Muscular safety was assessed using the following preferred terms and laboratory abnormalities: myalgia, muscle spasms, pain in extremity, muscular weakness (system organ class: musculoskeletal and connective tissue disorders), and creatine kinase \>5 ULN (repeated and confirmed). The percentage of unique participants is reported in the "Overall muscular disorder AESIs" category; a participant could have been represented in more than one of the individual muscular disorder AESIs.

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Neurocognitive Disorder

    Treatment-emergent AESIs were predefined and monitored throughout the study. Neurocognitive disorder was assessed using the following preferred terms: memory impairment, amnesia, and cognitive disorder (system organ class: nervous system disorders); confusional state and disorientation (system organ class: psychiatric disorders). The percentage of unique participants is reported in the "Overall neurocognitive disorder AESIs" category; a participant could have been represented in more than one of the individual neurocognitive disorder AESIs.

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: New Onset or Worsening Diabetes Mellitus

    Treatment-emergent AESIs were predefined and monitored throughout the study. New onset or worsening diabetes was assessed using the following preferred terms: type 2 diabetes mellitus, diabetes mellitus, hyperglycaemia, glucose tolerance impaired, diabetes mellitus inadequate control, and impaired fasting glucose (system organ class: metabolism and nutrition disorders); blood glucose increased, glycosylated haemoglobin increased, blood glucose abnormal, and glucose urine present (system organ class: investigations); and glycosuria (system organ class: renal and urinary disorders). The percentage of unique participants is reported in the "Overall new onset/worsening diabetes mellitus AESIs" category; a participant could have been represented in more than one of the individual new onset/worsening diabetes mellitus AESIs.

    Up to approximately 52 weeks

  • Percentage of Participants With the Indicated Event of Special Interest: Renal Disorder

    Treatment-emergent AESIs were predefined and monitored throughout the study. TEAEs potentially related to renal events were assessed using the following preferred terms: renal failure, renal impairment, acute kidney injury (system organ class: renal and urinary disorders); blood creatinine increased, glomerular filtration rate decreased, blood urea increased, estimated glomerular filtration rate (eGFR) \<30 milliliter per minute per 1.73 square meter (ml/min/1.73m\^2), and change from baseline in creatinine \>1 mg/dL (system organ class: investigations); and gout (system organ class: metabolism and nutrition disorders). The percentage of unique participants is reported in the "Overall renal disorder AESIs" category; a participant could have been represented in more than one of the individual renal disorder AESIs.

    Up to approximately 52 weeks

  • Change From Baseline to Week 52 in Uric Acid (Urate) Level

    Blood samples were drawn at defined time points during the course of the study to monitor uric acid (urate) levels.

    Baseline and Week 52

  • Change From Baseline to Week 52 in Creatinine Level

    Blood samples were drawn at defined time points during the course of the study to monitor creatinine levels.

    Baseline and Week 52

  • Change From Baseline to Week 52 in Hemoglobin Level

    Blood samples were drawn at defined time points during the course of the study to monitor hemoglobin levels.

    Baseline and Week 52

Secondary Outcomes (2)

  • Percent Change From Baseline to Week 12 in Low-density Lipoprotein Cholesterol (LDL-C)

    Baseline; Week 12

  • Absolute Change From Baseline to Week 12 in LDL-C

    Baseline; Week 12

Other Outcomes (15)

  • Percent Change From Baseline to Week 24 in LDL-C

    Baseline; Week 24

  • Percent Change From Baseline to Week 12 in Non-high-density Lipoprotein Cholesterol (Non-HDL-C)

    Baseline; Week 12

  • Percent Change From Baseline to Week 12 in Total Cholesterol (TC)

    Baseline; Week 12

  • +12 more other outcomes

Study Arms (2)

ETC-1002

EXPERIMENTAL

ETC-1002 180 mg/day

Drug: ETC-1002

Placebo

PLACEBO COMPARATOR

Placebo control

Drug: Placebo

Interventions

ETC-1002 180 mg tablets taken orally, once per day. Patients remain on ongoing statin therapy (not study provided)

Also known as: bempedoic acid
ETC-1002

Matching placebo tablets taken orally, once per day. Patients remain on ongoing statin therapy (not study provided)

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Fasting LDL-C ≥ 70 mg/dL
  • High cardiovascular risk (diagnosis of HeFH or ASCVD)
  • Be on maximally tolerated lipid-modifying therapy

You may not qualify if:

  • Total fasting triglyceride ≥500 mg/dL
  • Renal dysfunction or nephrotic syndrome or history of nephritis
  • Body Mass Index (BMI) ≥50kg/m2
  • Significant cardiovascular disease or cardiovascular event in the past 3 months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (104)

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Huntsville, Alabama, United States

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Cottonwood, Arizona, United States

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Phoenix, Arizona, United States

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Canoga Park, California, United States

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Santa Rosa, California, United States

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Bridgeport, Connecticut, United States

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Hartford, Connecticut, United States

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Atlantis, Florida, United States

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Boca Raton, Florida, United States

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Crestview, Florida, United States

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Crystal River, Florida, United States

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Daytona Beach, Florida, United States

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Lake Worth, Florida, United States

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Miami Lakes, Florida, United States

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Tampa, Florida, United States

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Park Ridge, Illinois, United States

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Iowa City, Iowa, United States

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Overland Park, Kansas, United States

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Covington, Kentucky, United States

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Louisville, Kentucky, United States

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Minden, Louisiana, United States

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Monroe, Louisiana, United States

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Shreveport, Louisiana, United States

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Auburn, Maine, United States

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Midland, Michigan, United States

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Saginaw, Michigan, United States

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Saint Cloud, Minnesota, United States

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Tupelo, Mississippi, United States

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Jefferson City, Missouri, United States

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Nashua, New Hampshire, United States

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Bridgewater, New Jersey, United States

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Cary, North Carolina, United States

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Mount Airy, North Carolina, United States

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Raleigh, North Carolina, United States

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Rocky Mount, North Carolina, United States

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Wilmington, North Carolina, United States

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Cincinnati, Ohio, United States

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Sandusky, Ohio, United States

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Willoughby, Ohio, United States

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Hillsboro, Oregon, United States

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Fort Worth, Texas, United States

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Houston, Texas, United States

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Katy, Texas, United States

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Kingwood, Texas, United States

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Orem, Utah, United States

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Richmond, Virginia, United States

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Puyallup, Washington, United States

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Tacoma, Washington, United States

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Vancouver, British Columbia, Canada

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Victoria, British Columbia, Canada

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Gatineau, Ontario, Canada

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Mississauga, Ontario, Canada

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Oshawa, Ontario, Canada

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Peterborough, Ontario, Canada

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Scarborough Village, Ontario, Canada

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Chicoutimi, Quebec, Canada

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Gatineau, Quebec, Canada

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Longueuil, Quebec, Canada

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Montreal, Quebec, Canada

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Québec, Quebec, Canada

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Saint-Charles-Borromée, Quebec, Canada

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Saint-Jean-sur-Richelieu, Quebec, Canada

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Berlin, Germany

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Bochum, Germany

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Dresden, Germany

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Essen, Germany

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Frankfurt, Germany

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Leipzig, Germany

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München, Germany

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Amsterdam, Netherlands

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Arnhem, Netherlands

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Eindhoven, Netherlands

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Goes, Netherlands

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Groningen, Netherlands

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Hardenberg, Netherlands

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Leiden, Netherlands

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Rotterdam, Netherlands

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Tilburg, Netherlands

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Venlo, Netherlands

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Zutphen, Netherlands

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Gdansk, Poland

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Gdynia, Poland

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Katowice, Poland

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Krakow, Poland

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Lodz, Poland

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Lowicz, Poland

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Poznan, Poland

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Puławy, Poland

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Torun, Poland

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Wroclaw, Poland

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Bexhill-on-Sea, United Kingdom

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Birmingham, United Kingdom

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Cardiff, United Kingdom

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Chesterfield, United Kingdom

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Chichester, United Kingdom

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Chippenham, United Kingdom

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Chorley, United Kingdom

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Glasgow, United Kingdom

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Hexham, United Kingdom

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Hull, United Kingdom

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Ipswich, United Kingdom

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Liverpool, United Kingdom

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Manchester, United Kingdom

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Reading, United Kingdom

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Related Publications (9)

  • Pinkosky SL, Newton RS, Day EA, Ford RJ, Lhotak S, Austin RC, Birch CM, Smith BK, Filippov S, Groot PHE, Steinberg GR, Lalwani ND. Liver-specific ATP-citrate lyase inhibition by bempedoic acid decreases LDL-C and attenuates atherosclerosis. Nat Commun. 2016 Nov 28;7:13457. doi: 10.1038/ncomms13457.

    PMID: 27892461BACKGROUND
  • Stone NJ, Robinson JG, Lichtenstein AH, Bairey Merz CN, Blum CB, Eckel RH, Goldberg AC, Gordon D, Levy D, Lloyd-Jones DM, McBride P, Schwartz JS, Shero ST, Smith SC Jr, Watson K, Wilson PW, Eddleman KM, Jarrett NM, LaBresh K, Nevo L, Wnek J, Anderson JL, Halperin JL, Albert NM, Bozkurt B, Brindis RG, Curtis LH, DeMets D, Hochman JS, Kovacs RJ, Ohman EM, Pressler SJ, Sellke FW, Shen WK, Smith SC Jr, Tomaselli GF; American College of Cardiology/American Heart Association Task Force on Practice Guidelines. 2013 ACC/AHA guideline on the treatment of blood cholesterol to reduce atherosclerotic cardiovascular risk in adults: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines. Circulation. 2014 Jun 24;129(25 Suppl 2):S1-45. doi: 10.1161/01.cir.0000437738.63853.7a. Epub 2013 Nov 12. No abstract available.

    PMID: 24222016BACKGROUND
  • Goldberg AC, Hopkins PN, Toth PP, Ballantyne CM, Rader DJ, Robinson JG, Daniels SR, Gidding SS, de Ferranti SD, Ito MK, McGowan MP, Moriarty PM, Cromwell WC, Ross JL, Ziajka PE; National Lipid Association Expert Panel on Familial Hypercholesterolemia. Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol. 2011 Jun;5(3 Suppl):S1-8. doi: 10.1016/j.jacl.2011.04.003. Epub 2011 Apr 12.

    PMID: 21600525BACKGROUND
  • Pollex RL, Joy TR, Hegele RA. Emerging antidyslipidemic drugs. Expert Opin Emerg Drugs. 2008 Jun;13(2):363-81. doi: 10.1517/14728214.13.2.363.

    PMID: 18537526BACKGROUND
  • Sharrett AR, Ballantyne CM, Coady SA, Heiss G, Sorlie PD, Catellier D, Patsch W; Atherosclerosis Risk in Communities Study Group. Coronary heart disease prediction from lipoprotein cholesterol levels, triglycerides, lipoprotein(a), apolipoproteins A-I and B, and HDL density subfractions: The Atherosclerosis Risk in Communities (ARIC) Study. Circulation. 2001 Sep 4;104(10):1108-13. doi: 10.1161/hc3501.095214.

    PMID: 11535564BACKGROUND
  • Ray KK, Bays HE, Catapano AL, Lalwani ND, Bloedon LT, Sterling LR, Robinson PL, Ballantyne CM; CLEAR Harmony Trial. Safety and Efficacy of Bempedoic Acid to Reduce LDL Cholesterol. N Engl J Med. 2019 Mar 14;380(11):1022-1032. doi: 10.1056/NEJMoa1803917.

  • Ridker PM, Lei L, Ray KK, Ballantyne CM, Bradwin G, Rifai N. Effects of bempedoic acid on CRP, IL-6, fibrinogen and lipoprotein(a) in patients with residual inflammatory risk: A secondary analysis of the CLEAR harmony trial. J Clin Lipidol. 2023 Mar-Apr;17(2):297-302. doi: 10.1016/j.jacl.2023.02.002. Epub 2023 Feb 14.

  • Ballantyne CM, Bays HE, Louie MJ, Smart J, Zhang Y, Ray KK. Factors Associated With Enhanced Low-Density Lipoprotein Cholesterol Lowering With Bempedoic Acid. J Am Heart Assoc. 2022 Aug 2;11(15):e024531. doi: 10.1161/JAHA.121.024531. Epub 2022 Aug 2.

  • Banach M, Duell PB, Gotto AM Jr, Laufs U, Leiter LA, Mancini GBJ, Ray KK, Flaim J, Ye Z, Catapano AL. Association of Bempedoic Acid Administration With Atherogenic Lipid Levels in Phase 3 Randomized Clinical Trials of Patients With Hypercholesterolemia. JAMA Cardiol. 2020 Oct 1;5(10):1124-1135. doi: 10.1001/jamacardio.2020.2314.

Related Links

MeSH Terms

Conditions

HypercholesterolemiaHyperlipidemiasAtherosclerosis

Interventions

8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid

Condition Hierarchy (Ancestors)

DyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesArteriosclerosisArterial Occlusive DiseasesVascular DiseasesCardiovascular Diseases

Results Point of Contact

Title
Medical Director
Organization
Esperion Therapeutics, Inc.

Study Officials

  • Ron Haberman, MD

    Esperion Therapeutics, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 20, 2016

First Posted

January 28, 2016

Study Start

January 21, 2016

Primary Completion

February 21, 2018

Study Completion

March 28, 2018

Last Updated

May 11, 2020

Results First Posted

April 20, 2020

Record last verified: 2020-04

Locations