Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM

NCT ID: NCT06081894

Last Updated: 2025-10-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

500 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-30

Study Completion Date

2026-09-30

Brief Summary

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This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

Detailed Description

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Conditions

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Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Aficamten

Participants in this arm will receive a single daily oral dose of 5 mg, 10 mg, 15 mg, or 20 mg of aficamten with dose levels guided by echocardiography assessments, for up to 72 weeks.

Group Type EXPERIMENTAL

Aficamten

Intervention Type DRUG

Oral Tablet

Placebo

Participants in this arm will receive placebo, for up to 72 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Oral Tablet

Interventions

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Aficamten

Oral Tablet

Intervention Type DRUG

Placebo

Oral Tablet

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Between 18-85 years of age
* Body mass index \< 40 kg/m2
* Diagnosed with nHCM and has a screening echocardiogram with the following:

* End-diastolic left ventricular (LV) wall thickness:

* ≥ 15 mm in one or more myocardial segments OR
* ≥ 13 mm in one or more wall segments and a known disease-causing gene mutation or positive family history of HCM AND
* Resting LVOT-G \< 30 mmHg AND Valsalva LVOT-G \< 50 mmHg AND
* LVEF ≥ 60%
* Participants with a history of intracavitary obstruction are eligible.
* NYHA class II or III
* Respiratory exchange ratio of ≥ 1.00 at screening by cardiopulmonary exercise testing (CPET) and predicted peak oxygen uptake (pVO2) ≤ 90% for age and sex
* KCCQ-CSS score of ≤ 85
* NT-proBNP of:

* NT-pro BNP ≥ 300 pg/mL or NT-proBNP ≥ 900 pg/mL if in atrial fibrillation or atrial flutter OR
* For Black participants, an NT-pro BNP ≥ 225 pg/mL or NT-proBNP ≥ 675 pg/mL if in atrial fibrillation or atrial flutter

Exclusion Criteria

* Significant valvular heart disease (per Investigator judgment)

* Moderate or severe valvular aortic stenosis or fixed subaortic obstruction
* Moderate or severe mitral regurgitation
* Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics nHCM (eg, Noonan syndrome, Fabry disease, amyloidosis)
* Known current unrevascularized coronary artery stenosis of ≥ 70% or documented history of myocardial infarction.
* History of LV systolic dysfunction (LVEF \< 45%) or stress cardiomyopathy
* Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations)
* Documented room air oxygen saturation reading \< 90% at screening or history of significant chronic obstructive pulmonary disease or severe/significant pulmonary hypertension
* History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia with exercise within 3 months prior to screening
* History of resistant hypertension (persistently elevated blood pressure despite maximal doses of 3 or more classes of medications for hypertension control)
* Screening diastolic blood pressure ≥ 100 mmHg
* Received prior treatment with aficamten
* Received treatment with mavacamten within 3 months prior to screening (must be discussed with the medical monitor prior to screening)
* Undergone septal reduction therapy \< 6 months prior to screening
* Is being considered for or is likely to be considered for heart transplant listing or left ventricular assist device placement during the study period
* Paroxysmal or permanent atrial fibrillation is excluded only if:

* rhythm restoring treatment (e.g., direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤ 3 months prior to screening
* rate control and anticoagulation have not been achieved for at least 3 months prior to screening.
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sanofi

INDUSTRY

Sponsor Role collaborator

Bayer

INDUSTRY

Sponsor Role collaborator

Cytokinetics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cytokinetics MD

Role: STUDY_DIRECTOR

Cytokinetics

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

UC San Diego Health - Sulpizio Cardiovascular Center

La Jolla, California, United States

Site Status

Keck Medical Center of USC (Outpatient Clinic)

Los Angeles, California, United States

Site Status

Cedars-Sinai Medical Center (Smidt Heart Institute)

Los Angeles, California, United States

Site Status

UCLA Medical Center Cardiovascular Clinic

Los Angeles, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Torrance, California, United States

Site Status

Yale New Haven Hospital

New Haven, Connecticut, United States

Site Status

MedStar Washington Hospital Center

Washington D.C., District of Columbia, United States

Site Status

Holy Cross Medical Group - Cardiology Associates

Fort Lauderdale, Florida, United States

Site Status

Investigational Drug Services, AdventHealth Orlando

Orlando, Florida, United States

Site Status

Emory Clinic

Atlanta, Georgia, United States

Site Status

Piedmont Heart Institute (CPET)

Fayetteville, Georgia, United States

Site Status

The Queen's Medical Center-Punchbowl

Honolulu, Hawaii, United States

Site Status

Northwestern University

Evanston, Illinois, United States

Site Status

Ascension St. Vincent

Indianapolis, Indiana, United States

Site Status

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

MedStar Union Memorial Hospital

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Lahey Hospital & Medical Center

Burlington, Massachusetts, United States

Site Status

Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

Henry Ford Hospital

Detroit, Michigan, United States

Site Status

M Health Fairview University of Minnesota Medical Center - East Bank

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Saint Luke's Hospital of Kansas City

Kansas City, Missouri, United States

Site Status

Morristown Medical Center

Morristown, New Jersey, United States

Site Status

Northwell Health North Shore University Hospital

Manhasset, New York, United States

Site Status

NYU Langone Health

New York, New York, United States

Site Status

Weill Cornell Medicine

New York, New York, United States

Site Status

Mount Sinai Hospital

New York, New York, United States

Site Status

Columbia University Medical Center/New York Presbyterian Hospital

New York, New York, United States

Site Status

Westchester Medical Center

Valhalla, New York, United States

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Sanger Heart & Vascular Institute - HCM Clinic

Charlotte, North Carolina, United States

Site Status

The Lindner Research Center at The Christ Hospital

Cincinnati, Ohio, United States

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University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Providence St. Vincent Medical Center

Portland, Oregon, United States

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Oregon Health & Science University

Portland, Oregon, United States

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Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

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UPMC Presbyterian

Pittsburgh, Pennsylvania, United States

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Medical University of South Carolina

Charleston, South Carolina, United States

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Greenville Memorial Hospital

Greenville, South Carolina, United States

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Ascension Saint Thomas Heart West

Nashville, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

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Baylor College of Medicine

Houston, Texas, United States

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Houston Methodist Hospital

Houston, Texas, United States

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Intermountain Medical Center

Murray, Utah, United States

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University of Utah Health

Salt Lake City, Utah, United States

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University of Virginia

Charlottesville, Virginia, United States

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Inova Health Care Services

Falls Church, Virginia, United States

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VCU Health Downtown Medical Campus

Richmond, Virginia, United States

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Carilion Clinic Cardiology

Roanoke, Virginia, United States

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Virginia Mason Medical Center

Seattle, Washington, United States

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Providence Sacred Heart Medical Center & Children's Hospital (CPET)

Spokane, Washington, United States

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WVU Medicine

Morgantown, West Virginia, United States

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University of Wisconsin Hospitals and Clinics

Madison, Wisconsin, United States

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Aurora St. Luke's Medical Center

Milwaukee, Wisconsin, United States

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Medical College of Wisconsin/Froedtert Hospital

Milwaukee, Wisconsin, United States

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Hospital Italiano de Buenos Aires Juan Domingo Peron

Buenos Aires, , Argentina

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ICBA - Instituto Cardiovascular de Buenos Aires

Buenos Aires, , Argentina

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Hospital Privado Centro Medico de Cordoba S.A

Córdoba, , Argentina

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The Prince Charles Hospital

Chermside, Queensland, Australia

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Flinders Medical Centre Cardiology Research

Adelaide, South Australia, Australia

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Fiona Stanley Hospital, AHF Unit

Murdoch, , Australia

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Instituto D'Or de Pesquisa e Ensino/Cardio Pulmonar da Bahia SA

Ondina, Salvador, Brazil

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Fundacao Felice Rocho / Hospital Felicio Rocho

Minas Gerais, , Brazil

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NUPEC Cardio / Cardio HVC

Minas Gerais, , Brazil

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CLINICOR Clinica Cardiologica LTDA

Ribeirão Preto, , Brazil

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Instituto Dante Pazzanese de Cardiologia

São Paulo, , Brazil

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Instituto do Coracao do Hospital de Aguiar

São Paulo, , Brazil

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Associacao Lar Sao Francisco de Assis na Providencia de Deus - Hospital Universitario Sao Francisco na Providencia de Deus (HUSF)

São Paulo, , Brazil

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Saraiva & Berlinger LTDA - EPP/IPECC - Instituto de Pesquisa Clinical de Campinas

São Paulo, , Brazil

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Hospital das Clinicas de Faculdade de Medicina de Ribeirao Preto da Universidade de Sao Paulo - HCFMRP

São Paulo, , Brazil

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Royal Alexandra Hospital

Edmonton, Alberta, Canada

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St. Boniface Hospital

Winnipeg, Manitoba, Canada

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Health Sciences North

Greater Sudbury, Ontario, Canada

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Montreal Heart Institute

Montreal, Quebec, Canada

Site Status

QEII Health Science Centre (Halifax Infirmary site)

Halifax, , Canada

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The 1st Affiliated Hospital of Sun Yat-sen University

Guangzhou, Guangdong, China

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Peking University First Hospital

Beijing, , China

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Fuwai Hospital, CAMS & PUMC

Beijing, , China

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Guangdong Provincial People's Hospital

Guangzhou, , China

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The First Hospital of Jilin University

Jilin, , China

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Zhongda Hospital Southeast University

Nanjin, , China

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Shanghai Ruijin Hospital Luwan Branch

Shanghai, , China

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Zhongshan Hospital Fudan University

Shanghai, , China

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People's Hospital of Liaoning Province

Shenyang, , China

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Remin Hospital of Wuhan University

Wuhan, , China

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The 1st affi. Hospital of Xi'an Jiaotong University

Xi'an, , China

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The 1st affiliated Hospital of Zhengzhou University

Zhenzhou, , China

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Aarhus University Hospital

Aarhus, , Denmark

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Department of Cardiology Copenhagen University Hospital, Bispebjerg

Copenhagen, , Denmark

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Odense University Hospital, Department of Cardiology

Odense, , Denmark

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Assistance Publique Hopitaux de Marseille

Marseille, , France

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Clinique Du Millenaire

Montpellier, , France

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Centre Hospitalier Universitaire De Montpellier

Montpellier, , France

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Assistance Publique Hopitaux De Paris

Paris, , France

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Assistance Publique Hopitaux de Paris

Paris, , France

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Centre Hospitalier Universitaire De Bordeaux

Pessac, , France

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Centre Hospitalier Universitaire De Nantes

Saint-Herblain, , France

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Hopital Rangueil

Toulouse, , France

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Charite-Universitaetsmedizin Berlin Campus Virchow-Klinikum (CVK) Medizinische Klinik mit Schwerpunkt Kardiologie

Berlin, , Germany

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Herzzentrum der Universitatsmedizin Gottingen

Göttingen, , Germany

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Universitaesklinkum Heidelberg Klinik fuer Kardiologie, Angeiologie und Pneumologie

Heidelberg, , Germany

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Univeristy Hospital Jena Clinic for Internal Medicine I (Cardiology, Angiology, Internal Intensive Care Medicine)

Jena, , Germany

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Evangelismos General Hospital of Athens

Athens, , Greece

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"Hippokration" General Hospital of Athens

Athens, , Greece

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University General Hospital Attikon

Attiki, , Greece

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Onassis Cardiac Surgery Center

Kallithea, , Greece

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Semmelweis Egyetem Varosmajori Sziv- es Ergyogyaszati Klinika

Budapest, , Hungary

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University of Szeged

Szeged, , Hungary

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Landspitali University Hospital

Reykjavik, , Iceland

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The Barzilai University Medical Center 2 - Cardiology Division

Ashkelon, , Israel

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Division of Cardiology Hadassah Medical Center

Jerusalem, , Israel

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Meir Medical Center

Kfar Saba, , Israel

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The Chaim Sheba Medical Center

Ramat Gan, , Israel

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Ziv Medical Center

Safed, , Israel

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Cardiology Department IRCCS San Donato Hospital - University of Milan

San Donato Milanese, Milano, Italy

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UOC Cardiologica

Brescia, , Italy

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Azienda Ospedaliera Universitaria Careggi pad. 13 Clinica Medica II Plano Cardiologia

Florence, , Italy

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Cardiologia Universitaria - UTIC - Riabilitazione Cardiologica Azienda Ospedaliero Universitaria - Ospedali Riuniti

Foggia, , Italy

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Unita Operativa di Riabilitazione Cardiologica, Istituto Auxologico Italiano, IRCCS - Auxologico San Luca, Piazzale Brescia, 20

Milan, , Italy

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Dipartimento Cardio-toraco- vascolare, ASST Grande Ospedale Metropolitano, NiguardaPiazza Ospedale Maggiore, 3

Milan, , Italy

Site Status

Dipartimento di Emergenze Cardiovascolari, Medicina Clinica e dell'Invecchiamento, AOU Federico II

Napoli, , Italy

Site Status

Fondazione Toscana Gabriele Monasterio Per La ricerca Medica e Di Sanita Pubbliga (FTGM) Cardio-Toracic-UOC Cardiology and Cardiovasulcar Medicine San Cataldo Hospital at CNR

Pisa, , Italy

Site Status

Dipartimento Cardiopolmonare, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele

Roma, , Italy

Site Status

Azienda Sanitaria Universitaria Giuliano Isontina Trieste SC Cardiologia

Trieste, , Italy

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Kyushu University Hospital

Fukuoka, , Japan

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Kobe City Medical Center General Hospital

Hyōgo, , Japan

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Hyogo Prefectural Harima-Himeii General Medical Center

Hyōgo, , Japan

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University of Tsukuba Hospital

Ibaraki, , Japan

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SHOWA Medical University Fujigaoka Hospital

Kanagawa, , Japan

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Kochi Medical School Hospital

Kochi, , Japan

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National Hospital Organization Kyoto Medical Center

Kyoto, , Japan

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Mie University Hospital

Mie, , Japan

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National University Corporation Tohoku University Tohoku University Hospital

Miyagi, , Japan

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Shinshu University Hospital

Nagano, , Japan

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Osaka Metropolitan University Hospital

Osaka, , Japan

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Osaka General Medical Center

Osaka, , Japan

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National Cerebral and Cardiovascular Center

Osaka, , Japan

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Osaka Medical and Pharmaceutical University Hospital

Osaka, , Japan

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Kitasato University Hospital

Sagamihara, , Japan

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Saitama Medical University International Medical Center

Saitama, , Japan

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Hamamatsu University Hospital

Shizuoka, , Japan

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St. Luke's International Hospital

Tokyo, , Japan

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Nippon Medical School Hospital

Tokyo, , Japan

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The University of Tokyo Hospital

Tokyo, , Japan

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Tokyo Women's Medical University Hospital

Tokyo, , Japan

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Teikyo University Hospital

Tokyo, , Japan

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P. Debyelaan 25

Maastricht, HX, Netherlands

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Amsterdam UMC, location AMC

Amsterdam, , Netherlands

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Erasmus University Medical Center

Rotterdam, , Netherlands

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Kardio Brynow Sp. z o.o.

Katowice, , Poland

Site Status

Unidade Local de Saude do Alto Ave, E.P.E

Creixomil, Guimaraes, Portugal

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Unidade Local de Saude da Regiao de Aveiro, E.P.E.

Aveiro, , Portugal

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Unidade Local de Saude de Sao Jose, E.P.E, - Hospital de Santa Marta Servico de Cardiologia

Lisbon, , Portugal

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Hospital da Luz

Lisbon, , Portugal

Site Status

Hospital CUF Descobertas, Lisbon, Cadiology Department

Lisbon, , Portugal

Site Status

Complejo Hospitalario Universitario A Coruna

A Coruña, , Spain

Site Status

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

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Hospital Clinic i Provincial de Barcelona

Barcelona, , Spain

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Hospital General Universitario Gregorio Marañón (HGUGM), Calle del Doctor Esquerdo, 46.

Madrid, , Spain

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Hospital Clinico San Carlos

Madrid, , Spain

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Hospital Universitario Puerta de Hierro

Madrid, , Spain

Site Status

Hospital Universitario Virgen de la Victoria Campus Universitario de Teatinos

Málaga, , Spain

Site Status

Hospital Clinico Universitario Virgen de la

Murcia, , Spain

Site Status

Hospital Son Llatzer

Palma de Mallorca, , Spain

Site Status

Hospital Universitario de Salamanca

Salamanca, , Spain

Site Status

Hospital Universitario Virgen Macarena

Seville, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Hospital Alvaro Cunqueiro

Vigo, , Spain

Site Status

NHS Tayside Ninewells Hospital & Medical School

Dundee, Scotland, United Kingdom

Site Status

NHS Greater Glasgow and Clyde Queen Elizabeth University Hospital

Glasgow, , United Kingdom

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Liverpool Heart and Chest Hospital NHS Foundation Trust

Liverpool, , United Kingdom

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Barts Health NHS Trust St Bartholomew's Hospital

London, , United Kingdom

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St George's University Hospitals NHS Foundation Trust,

London, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

Oxford Centre for Clinical Magnetic Resonance Research (Ocmr)

Oxford, , United Kingdom

Site Status

Countries

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United States Argentina Australia Brazil Canada China Denmark France Germany Greece Hungary Iceland Israel Italy Japan Netherlands Poland Portugal Spain United Kingdom

Other Identifiers

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2023-505797-15-00

Identifier Type: OTHER

Identifier Source: secondary_id

CY 6033

Identifier Type: -

Identifier Source: org_study_id

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