Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
NCT ID: NCT03470545
Last Updated: 2021-10-04
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
251 participants
INTERVENTIONAL
2018-05-29
2020-05-06
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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mavacamten (MYK-461)
mavacamten
mavacamten capsules
Placebo
Placebo
placebo oral capsule
Interventions
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mavacamten
mavacamten capsules
Placebo
placebo oral capsule
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
* Diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines and satisfy both criteria:
* Has documented left ventricular ejection fraction (LVEF) ≥55%
* NYHA Class II or III
* Has documented oxygen saturation at rest ≥90% at Screening
* Is able to perform an upright CPET and has a respiratory exchange ratio (RER) ≥1.0 at Screening per central reading
Exclusion Criteria
* History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
* History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
* Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at Screening
* Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
* Treatment (within 14 days prior to Screening) or planned treatment during the study with disopyramide or ranolazine
* Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of β-blockers and calcium channel blockers
* LVOT gradient with Valsalva maneuver \<30 mmHg at Screening
* Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation \[ASA\]) within 6 months prior to Screening or plans to have either of these treatments during the study
* ICD placement within 2 months prior to Screening or planned ICD placement during the study
* Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation, procedures, or completion
* Prior treatment with cardiotoxic agents such as doxorubicin or similar
18 Years
ALL
No
Sponsors
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MyoKardia, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Information Team
Role: STUDY_DIRECTOR
MyoKardia, Inc.
Locations
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Mayo Clinic Arizona
Scottsdale, Arizona, United States
Cedars-Sinai Medical Center (Smidt Heart Institute)
Los Angeles, California, United States
UCSF School of Medicine
San Francisco, California, United States
Stanford University
Stanford, California, United States
Yale New Haven Hospital
New Haven, Connecticut, United States
Mayo Clinic Jacksonville - PPDS
Jacksonville, Florida, United States
Northwestern University
Chicago, Illinois, United States
University Of Iowa Hospitals And Clinics
Iowa City, Iowa, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Spectrum Health
Grand Rapids, Michigan, United States
Washington University School of Medicine
St Louis, Missouri, United States
NYU Langone Medical Center
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
Carolinas Medical Center
Charlotte, North Carolina, United States
Duke Cardiology at Southpoint
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
St. Luke's Cardiology Associates
Bethlehem, Pennsylvania, United States
University of Pennsylvania (Penn Heart and Vascular Center)
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center Presbyterian
Pittsburgh, Pennsylvania, United States
Methodist University Hospital
Memphis, Tennessee, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Houston Methodist Hospital
Houston, Texas, United States
University of Texas Houston Medical School
Houston, Texas, United States
Intermountain Medical Center
Salt Lake City, Utah, United States
University of Utah
Salt Lake City, Utah, United States
University of Virginia Health System
Charlottesville, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
UZ Antwerpen
Edegem, Antwerpen, Belgium
Onze-Lieve-Vrouwziekenhuis
Aalst, Oost-Vlaanderen, Belgium
Hôpital Erasme
Brussels, , Belgium
Vseobecna fakultni nemocnice v Praze
Prague, , Czechia
Institut Klinicke a Experimentalni Mediciny
Prague, , Czechia
Aarhus Universitetshospital
Aarhus N, , Denmark
Bispebjerg Hospital
København NV, , Denmark
Odense Universitetshospital
Odense, , Denmark
CHRU Nantes
Nantes, Loire-Atlantique, France
Groupe Hospitalier Pitié Salpétrière
Paris, , France
Hôpital Européen Georges Pompidou
Paris, , France
Hôpital de Rangueil
Toulouse, , France
University Medicine Göttingen
Göttingen, Neidersachsen, Germany
Kerckhoff-Klinik-Forschungs-GmbH
Bad Nauheim, , Germany
Charité Campus Buch - Experimental and Clinical Research Center
Berlin, , Germany
Charité - Universitätsmedizin Berlin
Berlin, , Germany
Cardiologicum Dresden und Pirna
Dresden, , Germany
University Clinic Heidelberg - PPDS
Heidelberg, , Germany
Universitatsklinikum Schleswig-Holstein
Kiel, , Germany
Tel Aviv Sourasky Medical Center
Tel Aviv, Tel Aviv, Israel
Barzilai Medical Center
Ashkelon, , Israel
Hadassah Medical Center PPDS -
Jerusalem, , Israel
Rabin Medical Center - PPDS
Petah Tikva, , Israel
The Chaim Sheba Medical Center - The Edmond and Lily Safra Children's Hospital
Ramat Gan, , Israel
Kaplan Medical Center
Rehovot, , Israel
ZIV Medical Center
Safed, , Israel
Azienda Ospedaliera Universitaria Careggi
Florence, , Italy
Maastricht University Medical Center
Maastricht, Limburg, Netherlands
Erasmus MC
Rotterdam, South Holland, Netherlands
Collegium Medicum Uniwersytetu Jagiellonskiego
Krakow, Lesser Poland Voivodeship, Poland
Kardio Klinika Brynów
Katowice, Silesian Voivodeship, Poland
Szpital Kliniczny Przemienienia Panskiego Uniwesytetu Medycznego im. Karola Marcinkowskiego
Poznan, , Poland
Instytut Kardiologii im Prymasa Tysiaclecia Kardynala Stefana Wyszynskiego
Warsaw, , Poland
Hospital Garcia de Orta
Almada, , Portugal
Hospital da Luz
Lisbon, , Portugal
Hospital Universitario Virgen de La Arrixaca
El Palmar, Murcia, Spain
Hospital Universitario A Coruña
A Coruña, , Spain
Hospital Universitario Puerta de Hierro - Majadahonda
Madrid, , Spain
Hospital Universitario Ramon y Cajal
Madrid, , Spain
Hospital Universitario Virgen Macarena
Seville, , Spain
University Hospital of Wales
Cardiff, South Glamergon, United Kingdom
St Bartholomew's Hospital
London, , United Kingdom
Countries
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References
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Wang A, Lakdawala NK, Abraham TP, Nilles EK, Wojdyla DM, Owens AT, Bach RG, Saberi S, Sehnert A, Cresci S. Association Between age or Duration of Diagnosis in Obstructive Hypertrophic Cardiomyopathy and Response to Mavacamten Treatment: Exploratory Analysis of the EXPLORER-HCM Trial. J Card Fail. 2025 Jun;31(6):901-911. doi: 10.1016/j.cardfail.2024.10.449. Epub 2024 Dec 7.
Arnold SV, Gosch KL, Dolan C, Fine JT, Masri A, Saberi S, Wang A, Elliott PM, Hegde SM, Lam J, Sehnert AJ, Cresci S, Bach RG, Spertus JA. Association of Echocardiographic Parameters and Health Status in Patients With Obstructive Hypertrophic Cardiomyopathy: Insights From EXPLORER-HCM. Circulation. 2024 Nov 5;150(19):1560-1562. doi: 10.1161/CIRCULATIONAHA.123.067470. Epub 2024 Nov 4. No abstract available.
Garcia-Pavia P, Oreziak A, Masri A, Barriales-Villa R, Abraham TP, Owens AT, Jensen MK, Wojakowski W, Seidler T, Hagege A, Lakdawala NK, Wang A, Wheeler MT, Choudhury L, Balaratnam G, Shah A, Fox S, Hegde SM, Olivotto I. Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy. Eur Heart J. 2024 Dec 16;45(47):5071-5083. doi: 10.1093/eurheartj/ehae579.
Cresci S, Bach RG, Saberi S, Owens AT, Spertus JA, Hegde SM, Lakdawala NK, Nilles EK, Wojdyla DM, Sehnert AJ, Wang A. Effect of Mavacamten in Women Compared With Men With Obstructive Hypertrophic Cardiomyopathy: Insights From EXPLORER-HCM. Circulation. 2024 Feb 13;149(7):498-509. doi: 10.1161/CIRCULATIONAHA.123.065600. Epub 2023 Nov 14.
Wang A, Spertus JA, Wojdyla DM, Abraham TP, Nilles EK, Owens AT, Saberi S, Cresci S, Sehnert A, Lakdawala NK. Mavacamten for Obstructive Hypertrophic Cardiomyopathy With or Without Hypertension: Post-Hoc Analysis of the EXPLORER-HCM Trial. JACC Heart Fail. 2024 Mar;12(3):567-579. doi: 10.1016/j.jchf.2023.07.030. Epub 2023 Oct 18.
Wheeler MT, Olivotto I, Elliott PM, Saberi S, Owens AT, Maurer MS, Masri A, Sehnert AJ, Edelberg JM, Chen YM, Florea V, Malhotra R, Wang A, Oreziak A, Myers J. Effects of Mavacamten on Measures of Cardiopulmonary Exercise Testing Beyond Peak Oxygen Consumption: A Secondary Analysis of the EXPLORER-HCM Randomized Trial. JAMA Cardiol. 2023 Mar 1;8(3):240-247. doi: 10.1001/jamacardio.2022.5099.
Nassif M, Fine JT, Dolan C, Reaney M, Addepalli P, Allen VD, Sehnert AJ, Gosch K, Spertus JA. Validation of the Kansas City Cardiomyopathy Questionnaire in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Heart Fail. 2022 Aug;10(8):531-539. doi: 10.1016/j.jchf.2022.03.002. Epub 2022 May 4.
Reaney M, Addepalli P, Allen V, Spertus JA, Dolan C, Sehnert AJ, Fine JT. Longitudinal Psychometric Analysis of the Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) Using Outcomes from the Phase III EXPLORER-HCM Trial. Pharmacoecon Open. 2022 Jul;6(4):575-586. doi: 10.1007/s41669-022-00340-8. Epub 2022 Jun 20.
Hegde SM, Lester SJ, Solomon SD, Michels M, Elliott PM, Nagueh SF, Choudhury L, Zemanek D, Zwas DR, Jacoby D, Wang A, Ho CY, Li W, Sehnert AJ, Olivotto I, Abraham TP. Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2021 Dec 21;78(25):2518-2532. doi: 10.1016/j.jacc.2021.09.1381.
Xie J, Wang Y, Xu Y, Fine JT, Lam J, Garrison LP. Assessing health-related quality-of-life in patients with symptomatic obstructive hypertrophic cardiomyopathy: EQ-5D-based utilities in the EXPLORER-HCM trial. J Med Econ. 2022 Jan-Dec;25(1):51-58. doi: 10.1080/13696998.2021.2011301.
Burstein Waldman C, Owens A. A plain language summary of the EXPLORER-HCM study: mavacamten for obstructive hypertrophic cardiomyopathy. Future Cardiol. 2021 Oct;17(7):1269-1275. doi: 10.2217/fca-2021-0044. Epub 2021 May 21.
Spertus JA, Fine JT, Elliott P, Ho CY, Olivotto I, Saberi S, Li W, Dolan C, Reaney M, Sehnert AJ, Jacoby D. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): health status analysis of a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2021 Jun 26;397(10293):2467-2475. doi: 10.1016/S0140-6736(21)00763-7. Epub 2021 May 15.
Olivotto I, Oreziak A, Barriales-Villa R, Abraham TP, Masri A, Garcia-Pavia P, Saberi S, Lakdawala NK, Wheeler MT, Owens A, Kubanek M, Wojakowski W, Jensen MK, Gimeno-Blanes J, Afshar K, Myers J, Hegde SM, Solomon SD, Sehnert AJ, Zhang D, Li W, Bhattacharya M, Edelberg JM, Waldman CB, Lester SJ, Wang A, Ho CY, Jacoby D; EXPLORER-HCM study investigators. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020 Sep 12;396(10253):759-769. doi: 10.1016/S0140-6736(20)31792-X. Epub 2020 Aug 29.
Ho CY, Olivotto I, Jacoby D, Lester SJ, Roe M, Wang A, Waldman CB, Zhang D, Sehnert AJ, Heitner SB. Study Design and Rationale of EXPLORER-HCM: Evaluation of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy. Circ Heart Fail. 2020 Jun;13(6):e006853. doi: 10.1161/CIRCHEARTFAILURE.120.006853. Epub 2020 Jun 5.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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MYK-461-005
Identifier Type: -
Identifier Source: org_study_id
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