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This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment.
United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mavacamten | Participants will receive mavacamten as prescribed by a physician according to standard of care for symptomatic oHCM |
| |
| Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium channel blocker (CCB) / disopyramide | Participants will receive BB/non-DHP CCB/disopyramide as prescribed by a physician according to standard of care for symptomatic oHCM |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mavacamten | Drug | As per product label |
| |
| Non-mavacamten symptomatic oHCM therapy |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence rate of new or worsening heart failure due to systolic dysfunction among participants with symptomatic obstructive HCM during periods of exposure to mavacamten and other oHCM treatment | Systolic dysfunction defined as symptomatic Left Ventricular Ejection Fraction (LVEF) < 50%. United States participants only. | Up to 5 Years |
| Change in resting left ventricular outflow tract (LVOT) gradient from baseline | European Participants only | Up to 18 months |
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of arrhythmia | Incidence rates of participants with arrhythmia (new onset) | Baseline, and up to 18 months (European participants), or 5 Years (United States participants) |
| New or worsening heart failure due to systolic dysfunction |
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Inclusion Criteria
United States Sub-Study
Diagnosis of obstructive HCM consistent with 2020 American Heart Association/American College of Cardiology (AHA/ACC) guidelines.
Has documented LVEF of ≥ 55% recorded by echocardiography within the last 6 months.
Symptoms consistent with NYHA functional class II-IV.
Receiving beta blocker (BB)s, non-dihydropyridine calcium. channel blockers (nonDHP CCBs), disopyramide, and/or mavacamten (once available) as part of routine clinical care; or currently receiving no treatment due to intolerance or failure of prior treatment (eg, BBs, non-DHP CCBs, or disopyramide) for obstructive HCM.
European Sub-study
Exclusion Criteria
United States Sub-Study
European Sub-study
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The study population will include adult participants with symptomatic obstructive HCM who are receiving pharmacological treatment for obstructive HCM (for example, BBs, non-DHP CCBs, disopyramide, and/or mavacamten) as part of routine care in the United States or Europe.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| BMS Clinical Trials Contact Center www.BMSClinicalTrials.com | Contact | 855-907-3286 | Clinical.Trials@bms.com | |
| First line of the email MUST contain NCT # and Site #. | Contact |
| Name | Affiliation | Role |
|---|---|---|
| Bristol-Myers Squibb | Bristol-Myers Squibb | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Alaska Heart Institute | Recruiting | Anchorage | Alaska | 99508 | United States |
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| Label | URL |
|---|---|
| BMS Clinical Trial Information | View source |
| FDA Safety Alerts and Recalls | View source |
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| Drug |
Including Beta-blocker (BB), non-dihydropyridine (non-DHP) calcium channel blocker (CCB) and/or disopyramide as per product label |
|
Symptomatic left ventricular ejection fraction (LVEF) <50%. European Participants only
| Baseline and up to 18 months |
| Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 50 mmHG | European participants only | Baseline and up to 18 months |
| Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 30 mmHG | European participants only | Baseline and up to 18 months |
| Occurrence of Major Adverse Cardiovascular Events (MACE) | A composite endpoint consisting of non-fatal acute Myocardial Infarction (MI), stroke, hospitalization due to Heart Failure (HF) and Cardiovascular (CV) mortality. United States participants only. | Up to 5 Years |
| Occurrence of non-fatal acute Myocardial Infarction (MI) | United States participants only | Up to 5 Years |
| Occurrence of Stroke | United States participants only | Up to 5 Years |
| Occurrence of hospitalization due to heart failure | Baseline, and up to 18 months (European participants), or 5 Years (United States participants) |
| Occurrence of cardiovascular mortality | United States participants only | Up to 5 Years |
| Occurrence of all-cause mortality | Baseline, and up to 18 months (European participants), or 5 Years (United States participants) |
| Evaluation of functional responses: New York Heart Association (NYHA) function class | Percentage of Participants Whose NYHA Class Changes | Baseline, and up to 18 months (European participants), or 5 Years (United States participants) |
| Evaluation of functional responses: Left Ventricular Outflow Tract (LVOT) gradient | Mean Change From Baseline in LVOT Gradients At Rest and Provoked | Baseline, and up to 18 months (European participants), or 5 Years (United States participants) |
| Evaluation of functional responses: LVEF | Mean Change from Baseline in LVEF | Baseline, and up to 18 months (European participants), or 5 Years (United States participants) |
| Evaluation of patient reported outcome measure: Kansas City Cardiomyopathy Questionnaire 23 (KCCQ-23) | The KCCQ-23 is a disease-specific health status instrument composed of 23 items that quantifies the domains of physical limitations, symptoms, self-efficacy, social limitation and quality of life from heart failure. Scores range from 0-100, in which higher scores reflect better health status. United States participants only | Baseline, and up to 5 Years |
| Evaluation of patient reported outcome measure: EuroQol Five Dimensions Questionnaire (5-level) (EQ-5D-5L): Health Utility Index | The EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L health utility index and EQ Visual Analog Scale (VAS). EQ-5D-5L health utility index is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each has 5 levels of perceived problems (1-no problem, 2-slight problems, 3-moderate problems, 4-severe problems, 5-extreme problems). Participants select an answer for each of the 5 dimensions that best matches his/her health "today". Responses are used to generate a weighted summary index, which ranges from 0 to 1.00. A higher score indicates better health and positive changes from baseline indicate improvement of health. United States participants only | Baseline, and up to 5 Years |
| Evaluation of patient reported outcome measure: EQ-5D-5L: VAS | The EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and VAS. The VAS self-rating records the participant's own assessment of his or her overall health status at the time of completion, on a vertical line VAS with scale of 0 (the worst health you can imagine) to 100 (the best health you can imagine). A higher score indicates better health and positive changes from baseline indicate improvement of health status. United States participants only | Baseline, and up to 5 Years |
| Evaluation of biomarkers of response: NT-proBNP | Change From Baseline in Serum concentration of NT-proBNP. United States participants only | Baseline, and up to 5 Years |
| Evaluation of biomarkers of response: Cardiac troponin | Change From Baseline in Serum Concentration of Cardiac Troponins. United States participants only | Baseline, and up to 5 Years |
| Pima Heart and Vascular | Recruiting | Tucson | Arizona | 85704 | United States |
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| UAMS | Recruiting | Little Rock | Arkansas | 72205-7199 | United States |
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| UC San Diego School of Medicine | Recruiting | La Jolla | California | 92037 | United States |
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| Keck School of Medicine of USC-Usc | Recruiting | Los Angeles | California | 90033 | United States |
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| Stanford Health Care Hospital & Clinics | Recruiting | Palo Alto | California | 94304 | United States |
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| University Of California San Francisco Medical Center | Recruiting | San Francisco | California | 94117 | United States |
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| UC Denver, AMC | Recruiting | Aurora | Colorado | 48377 | United States |
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| Hartford HealthCare | Recruiting | Hartford | Connecticut | 06106 | United States |
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| MedStar Washington Hospital Center | Recruiting | Washington D.C. | District of Columbia | 20010 | United States |
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| Uf Health Jacksonville | Recruiting | Jacksonville | Florida | 32209 | United States |
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| University of Hawaii Cancer Center | Recruiting | Honolulu | Hawaii | 96813 | United States |
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| University of Chicago Dept of Medicine | Recruiting | Chicago | Illinois | 60637 | United States |
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| Indiana. University | Recruiting | Indianapolis | Indiana | 46202 | United States |
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| Franciscan Physician Network-Indiana Heart Physicians | Recruiting | Indianapolis | Indiana | 46237 | United States |
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| University of Iowa Hospitals & Clinics | Recruiting | Iowa City | Iowa | 52242 | United States |
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| University Of Kansas Medical Center & Medical Pavilion | Recruiting | Kansas City | Kansas | 66160 | United States |
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| University Of Maryland, Ihv | Recruiting | Baltimore | Maryland | 21201 | United States |
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| MedStar Health Research Institute | Recruiting | Baltimore | Maryland | 21218 | United States |
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| Harvard (Massachusetts General Hospital) | Recruiting | Boston | Massachusetts | 02114 | United States |
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| Harvard Medical School - Brigham and Women's Hospital (BWH) | Recruiting | Boston | Massachusetts | 02215 | United States |
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| Spectrum Health Medical Group | Recruiting | Grand Rapids | Michigan | 49525 | United States |
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| Henry Ford Health System | Recruiting | West Bloomfield | Michigan | 48322 | United States |
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| Minneapolis Heart Institute Foundation | Recruiting | Minneapolis | Minnesota | 55407 | United States |
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| Cardiology Associates Research, Llc | Recruiting | Tupelo | Mississippi | 38801 | United States |
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| St. Luke's Mid-America Heart Institute | Recruiting | Kansas City | Missouri | 64111 | United States |
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| Washington University | Recruiting | St Louis | Missouri | 63110 | United States |
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| CHI Health Reseach Center | Recruiting | Omaha | Nebraska | 68124 | United States |
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| Hackensack University Medical Center | Recruiting | Hackensack | New Jersey | 07601 | United States |
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| Morristown Medical Center | Recruiting | Morristown | New Jersey | 07960 | United States |
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| Bassett Medical Center | Recruiting | Cooperstown | New York | 13326 | United States |
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| Northwell Health | Recruiting | Manhasset | New York | 11030 | United States |
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| Mount Sinai West | Recruiting | New York | New York | 10019 | United States |
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| Mount Sinai Hospital | Recruiting | New York | New York | 10029 | United States |
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| Saint Francis Hospital | Recruiting | Roslyn | New York | 11576 | United States |
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| WMCHealth Advanced Physician Services | Recruiting | Valhalla | New York | 10595 | United States |
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| Focus Clinical Research | Recruiting | Charlotte | North Carolina | 28207 | United States |
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| Duke University Health System | Recruiting | Durham | North Carolina | 27710 | United States |
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| Christ Hospital Health Network | Recruiting | Cincinnati | Ohio | 45219 | United States |
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| University Hospitals Cleveland Medical Center | Recruiting | Cleveland | Ohio | 44106 | United States |
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| Cleveland Clinic | Recruiting | Cleveland | Ohio | 44195 | United States |
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| St. Luke's University Health Network | Recruiting | Bethlehem | Pennsylvania | 18015 | United States |
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| Lancaster General Hospital | Recruiting | Lancaster | Pennsylvania | 17603 | United States |
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| AHN Allegheny General Hospital | Recruiting | Pittsburgh | Pennsylvania | 15212 | United States |
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| University Of Pittsburgh | Recruiting | Pittsburgh | Pennsylvania | 15213 | United States |
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| Geisinger Health System | Recruiting | Wilkes-Barre | Pennsylvania | 18711 | United States |
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| Prisma Health - Upstate | Recruiting | Greenville | South Carolina | 29605 | United States |
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| Tristar Centennial Medical Ctr | Recruiting | Nashville | Tennessee | 37203 | United States |
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| Saint Thomas Health | Recruiting | Nashville | Tennessee | 37205-2018 | United States |
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| North Texas Cardiology Center | Recruiting | Dallas | Texas | 75208 | United States |
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| Baylor Scott & White Research Institute | Recruiting | Dallas | Texas | 75246 | United States |
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| The Methodist Hospital, Methodist Cancer Cencer | Recruiting | Houston | Texas | 77030 | United States |
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| BI Research Center | Recruiting | Houston | Texas | 77084 | United States |
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| Baylor Scott & White The Heart Hospital Plano | Recruiting | Plano | Texas | 75093 | United States |
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| Baylor Scott & White Medical Centre - Temple | Recruiting | Temple | Texas | 76508 | United States |
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| The Rector and Visitors of the Univ of Virginia | Recruiting | Charlottesville | Virginia | 22908 | United States |
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| VCU Medical Center | Recruiting | Richmond | Virginia | 23298 | United States |
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| Carilion Clinic; Virginia Tech-Carilion School of Medicine | Recruiting | Roanoke | Virginia | 24014 | United States |
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| Virginia Mason Medical Center | Recruiting | Seattle | Washington | 98101 | United States |
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| WVU Hospitals | Recruiting | Morgantown | West Virginia | 26506 | United States |
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| University Of Wisconsin - Madison | Recruiting | Madison | Wisconsin | 53792 | United States |
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| Medical College of Wisconsin, Inc (PI Address) | Recruiting | Milwaukee | Wisconsin | 53226 | United States |
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| KH St.Josef Braunau am Inn | Recruiting | Braunau am Inn | 5280 | Austria |
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| LKH-Univ. Klinikum Graz | Recruiting | Graz | 8036 | Austria |
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| Local Institution - 0088 | Not yet recruiting | Innsbruck | 6020 | Austria |
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| Local Institution - 0084 | Withdrawn | Klagenfurt | 9020 | Austria |
| Kepler Universitätsklinikum Linz | Recruiting | Linz | 4021 | Austria |
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| Local Institution - 0085 | Not yet recruiting | Sankt Pölten | 3100 | Austria |
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| Local Institution - 0083 | Not yet recruiting | Vienna | 1090 | Austria |
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| Centre Hospitalier Haguenau | Recruiting | Haguenau | Bas Rhin | 67500 | France |
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| CHU Nantes Hopital Nord Laennec | Recruiting | Saint-Herblain | Loire Atlantique | 44800 | France |
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| Hôpital Saint Vincent-de-Paul | Recruiting | Lille | Nord | 59000 | France |
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| CHU Amiens-Picardie | Recruiting | Amiens | Somme | 80054 | France |
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| Hôpital Sainte Musse | Recruiting | Toulon | Var | 83000 | France |
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| CHU De Poitiers - Hopital De La Miletrie | Recruiting | Poitiers | Vienne | 86000 | France |
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| Local Institution - 0117 | Withdrawn | Montpellier | 34000 | France |
| Ostalb-Klinikum Aalen | Recruiting | Aalen | Baden-Wurttemberg | 73430 | Germany |
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| Local Institution - 0101 | Withdrawn | Erlangen | Bavaria | 91054 | Germany |
| Universitatsklinikum Muenster | Recruiting | Münster | North Rhine-Westphalia | 48149 | Germany |
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| Praxisklinik Herz Und Gefasse-Klinikum Weisser Hirsch | Recruiting | Dresden | Saxony | 01099 | Germany |
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| Universitaetsklinikum Leipzig Aoer | Recruiting | Leipzig | Saxony | 04103 | Germany |
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| Immanuel Klinikum Bernau | Recruiting | Bernau | State of Berlin | 16321 | Germany |
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| Kardiologische und angiologische Praxen im Spreebogen | Recruiting | Berlin | 10719 | Germany |
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| Research & Cardiovascular Corp | Recruiting | Ponce | 00717-1322 | Puerto Rico |
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| Hospital Fundacion Son Llatzer | Recruiting | Palma de Mallorca | Balearic Islands | 07198 | Spain |
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| Hospital Universitario Puerta de Hierro Majadahonda | Recruiting | Majadahonda | Madrid | 28222 | Spain |
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| Hospital Universitario Virgen de La Arrixaca | Recruiting | El Palmar | Murcia | 30120 | Spain |
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| Hospital Universitario Central de Asturias | Recruiting | Oviedo | Principality of Asturias | 33011 | Spain |
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| Hospital Universitario de Burgos | Recruiting | Burgos | 09006 | Spain |
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| Local Institution - 0102 | Withdrawn | Madrid | 28007 | Spain |
| Hospital Clinico San Carlos | Recruiting | Madrid | 28040 | Spain |
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| Hospital Clinico Universitario Virgen de La Victoria | Recruiting | Málaga | 29010 | Spain |
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| Local Institution - 0113 | Not yet recruiting | London | Greater London | SW3 6NP | United Kingdom |
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| Imperial College Healthcare NHS Trust | Recruiting | London | Greater London | W6 8RF | United Kingdom |
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| University Of Manchester | Recruiting | Manchester | Greater Manchester | M13 9WL | United Kingdom |
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| Liverpool Heart and Chest Hospital | Recruiting | Liverpool | Merseyside | L14 3PE | United Kingdom |
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| University Hospitals Birmingham NHS Foundation Trust | Recruiting | Birmingham | West Midlands | B15 2GW | United Kingdom |
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| Leeds General Infirmary | Recruiting | Leeds | West Yorkshire | LS1 3EX | United Kingdom |
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| University Hospital of Wales | Recruiting | Cardiff | CF14 4XW | United Kingdom |
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| ID | Term |
|---|---|
| D002312 | Cardiomyopathy, Hypertrophic |
| D006333 | Heart Failure |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001020 | Aortic Stenosis, Subvalvular |
| D001024 | Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |
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| ID | Term |
|---|---|
| C000605992 | MYK-461 |
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