New pill aims to help heart patients breathe easier and move more

NCT ID NCT05186818

Disease control Sponsor: Cytokinetics Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tested a daily pill called aficamten in 282 adults with a type of thickened heart muscle that blocks blood flow and causes symptoms like shortness of breath and chest pain. Participants received either aficamten or a placebo for 24 weeks. The main goal was to see if the drug improved exercise capacity, measured by peak oxygen uptake during a stress test. Secondary goals included assessing quality of life and symptoms using a standard questionnaire.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Aficamten (CK-3773274) – a daily oral tablet
What this could lead to: If it works, aficamten could become a new treatment option to improve exercise capacity and quality of life for people with obstructive hypertrophic cardiomyopathy.
What could go wrong: This is a completed Phase 3 trial, but results are not yet published. The drug may not show significant benefit over placebo, and side effects could limit its use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

282 people

The number who actually took part.

Started

Feb 2022

Finished

Dec 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 85 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Males and females between 18 and 85 years of age, inclusive, at screening. * Body mass index \<35 kg/m2. * Diagnosed with HCM per the following criteria: * Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and * Has an end-diastolic LV wall thickness as measured by the echocardiography core laboratory of: * ≥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 * Has resting LVOT-G ≥30 mmHg and post-Valsalva LVOT G ≥50 mmHg during screening as determined by the echocardiography core laboratory. * LVEF ≥60% at screening as determined by the echocardiography core laboratory. * New York Heart Association (NYHA) Functional Class II or III at screening. * Hemoglobin ≥10g/dL at screening. * Respiratory exchange ratio (RER) ≥1.05 and pVO2 ≤90% predicted on the screening CPET per the core laboratory. * Patients on beta-blockers, verapamil, diltiazem, or disopyramide should have been on stable doses for \>6 weeks prior to randomization and anticipate remaining on the same medication regimen during the trial. Patients treated with disopyramide must also be concomitantly treated with a beta blocker and/or calcium channel blocker. Key Exclusion Criteria: * Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics oHCM (eg, Noonan syndrome, Fabry disease, amyloidosis). * Significant valvular heart disease (per investigator judgment). * Moderate-severe valvular aortic stenosis. * Moderate-severe mitral regurgitation not due to systolic anterior motion of the mitral valve. * History of LV systolic dysfunction (LVEF \<45%) or stress cardiomyopathy at any time during their clinical course. * Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations). * Has been treated with septal reduction therapy (surgical myectomy or percutaneous alcohol septal ablation) or has plans for either treatment during the trial period. * Documented paroxysmal atrial fibrillation during the screening period. * Paroxysmal or permanent atrial fibrillation is only excluded IF: * rhythm restoring treatment (eg, direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤6 months prior to screening. * rate control and anticoagulation have not been achieved for at least 6 months prior to screening. * History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to screening. * Has received prior treatment with CK-3773274 or mavacamten.

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Conditions

The condition(s) this trial relates to.

hypertrophic cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Obstructive hypertrophic cardiomyopathy (OHCM)

Contacts and locations

Locations

Aalborg University Hospital, Department of Cardiology

Aalborg, 9100, Denmark
Alaska Heart and Vascular Institute

Anchorage, Alaska, 99508, United States
Amsterdam UMC, location AMC

Amsterdam, 1105, Netherlands
Ascension St. John Clinical Research Institute

Tulsa, Oklahoma, 74104, United States
Azienda Ospedaliero Universitaria Careggi Dipartimento Cardiotoracovascolare - Cardiomiopatie Unit

Florence, 50134, Italy
Barts Health NS Trust

London, EC1A 7BE, United Kingdom
Beijing Anzhen Hospital

Beijing, Beijing Municipality, 100029, China
Beijing Chao-Yang Hospital, Capital Medical University

Beijing, Beijing Municipality, 100020, China
Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States
CHU Pitie-Salpetriere, Centre de reference maladies cardiaques hereditaires ou rares

Paris, France
CHU de Bordeaux Hopital Cardiologique Haut-Leveque

Pessac, 33600, France
CHU de Marseille - Hopital de la Timone Service de cardiologie

Marseille, 13005, France
CHU de Nantes

Nantes, 44093, France
CHU du Haut Lveque Cardiologie

Pessac, 33604, France
Cedars-Sinai Medical Center - Smidt Heart Institute Clinic

Los Angeles, California, 90048, United States
Centro Hospitalar Do Baixo Vouga, EPE AV ARTUR RAVARA

Aveiro, 3814-501, Portugal
Charite-Universitaetsmedizin Berlin Campus Virchow-Klinikum (CVK) Medizinische Klinik mit Schwerpunkt Kardiologie

Berlin, 13353, Germany
Charles University

Prague, 128 08, Czechia
Cleveland Clinic

Cleveland, Ohio, 44195, United States
Columbia University Medical Center/NY Presbyterian Hospital

New York, New York, 10032, United States
Complejo Hospitalario Universitario

A Coruña, La Coruna, 15006, Spain
Copenhagen University Hospital

Copenhagen, 2400, Denmark
Department of Cardiology Aarhus University Hospital

Aarhus, 8200, Denmark
Dipartimento di Medicina Clinica e Molecolare Universita Sapienza di Roma Unita di Terapia Intensiva Cardiologica Azienda Ospedaliero Universitaria Sant'Andrea

Roma, 1035-39, Italy
Duke Health Center Arringdon

Morrisville, North Carolina, 27560, United States
Emory Clinic

Atlanta, Georgia, 30322, United States
Erasmus Medical Center

Rotterdam, 3015, Netherlands
Fondazione toscana Gabriele Monesterio per la ricarca medica Dipartimento Cardiotoracico UOC Cardiologia e Medicina Cardiovasculare Ospedale San Cataldo

Pisa, 56124, Italy
Froedtert Hospital

Milwaukee, Wisconsin, 53226, United States
Hadassah Medical Center- Ein Kerem

Jerusalem, 9112001, Israel
Henry Ford Medical Center

Detroit, Michigan, 48202, United States
Hjertecentret (The Heart Center)

Copenhagen, 2100, Denmark
Holy Cross Hospital / Cardiology Associates

Fort Lauderdale, Florida, 33308, United States
Hopital Lariboisiere, Service de Cardiologie

Paris, 75010, France
Hopital Universitaire de Rangueil (CHU de Toulouse) Service de Cardiologie

Toulouse, Cedex, 31059, France
Hospital Clinic de Barcelona

Barcelona, 08036, Spain
Hospital Clinico Universitario Virgen de la Arrixaca

Murcia, Spain
Hospital Universitario Puerta de Hierro

Madrid, Majadahonda, Spain
Hospital Universitario Son Llatzer Secretaria de Cardiologia

Palma, 07198, Spain
Hospital Universitario Virgen Macarena-merge

Seville, 41009, Spain
Hospital Universitario de Salamanca

Salamanca, 37007, Spain
Hospital da Luz

Lisbon, 1500-650, Portugal
Hospital of the University of Pennsylvania (University of Pennsylvania School of Medicine)

Philadelphia, Pennsylvania, 19104, United States
Houston Methodist Hospital

Houston, Texas, 77030, United States
Hôpital Européen Georges Pompidou

Paris, 75015, France
Interni klinika kardiologie a angiologie

Prague, 12808, Czechia
Kaplan Medical Center

Rehovot, 76100, Israel
Kardio Brynow S.C

Katowice, Poland
Kerckhoff-Klinik GmbH Abteilung Administration Forschung und Lehre

Bad Nauheim, 61231, Germany
Klinikumder Georg-August-Universitaet

Göttingen, 37075, Germany
Krakowskie Centrum

Krakow, Poland
Lahey Hospital & Medical Center

Burlington, Massachusetts, 01805, United States
Liverpool Heart and Chest Hospital - Liverpool Heart and Chest Hospital NHS Foundation Trust- HWS Greater Glasgow and Clyde

Liverpool, L14 3LB, United Kingdom
M Health Fairview University of Minnesota Medical Center - East Bank

Minneapolis, Minnesota, 55455, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Mayo Clinic

Rochester, Minnesota, 55905, United States
Mayo Clinic Florida

Jacksonville, Florida, 32224, United States
MedStar Medical Group Cardiology at Union Memorial

Baltimore, Maryland, 21218, United States
MedStar Washington Hospital Center

Washington D.C., District of Columbia, 20010, United States
Medstar Franklin Square Medical Center

Baltimore, Maryland, 21237, United States
Michigan Medicine

Ann Arbor, Michigan, 48109, United States
Morristown Medical Center

Morristown, New Jersey, 07960, United States
Mount Sinai Hospital

New York, New York, 10029, United States
NYU Langone Health

New York, New York, 10016, United States
Nanfang Hospital Southern Medical University

Guanzhou, Guangdong, 516006, China
Narodowy Instytut Kardiologii Stefana Kardynała Wyszynskiego- Panstwowy Instytut Badawczy

Warsaw, Masovian Voivodeship, 04-628, Poland
Northwestern University

Chicago, Illinois, 60614, United States
Oregon Health & Science University

Portland, Oregon, 97239, United States
Oxford Centre for Clinical Magnetic Resonance Research

Oxford, OX3 9DU, United Kingdom
Peking University Third Hospital

Beijing, Beijing Municipality, 10019, China
Piedmont Fayette Hospital

Fayetteville, Georgia, 30214, United States
Queen Elizabeth Hospital Birmingham University Hospitals Birmingham NHS Foundation Trust

Edgbaston, Birmingham, B15 2GW, United Kingdom
Queen Elizabeth University Hospital - HWS Greater Glasgow and Clyde

Glasgow, G1 4TF, United Kingdom
Radcliffe Department of Medicine

Oxford, OX3 9DU, United Kingdom
Royal Brompton Hospital

London, SW3 6NP, United Kingdom
Saint Luke's Hospital of Kansas City

Kansas City, Missouri, 64111, United States
Sanger Heart and Vascular Institute

Charlotte, North Carolina, 10595, United States
Sanger Heart and Vascular Institute - HCM Clinic

Charlotte, North Carolina, 28204, United States
Semmelweis Egyetem Varosmajori Sziv es Ergyogyasazati Klinika

Budapest, Hungary
Spectrum Health Medical Group Cardiovascular Medicine

Grand Rapids, Michigan, 49525, United States
St George's University Hospitals NHS Foundation Trust

London, SW17 0QT, United Kingdom
Stanford University Hospital

Stanford, California, 94305, United States
Stern Cardiovascular Foundation, Inc

Germantown, Tennessee, 38138, United States
The Barzilai University Medical Center

Ashkelon, 78100, Israel
The Barzilai University Medical Center

Ashkelon, 7830604, Israel
The First Affiliated Hospital of Xi'an Jiaotong University

Xi’an, Shanxi, 710061, China
The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, 450018, China
The First Hospital of Jinlin University

Changchun, Jinlin, 130031, China
The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States
The Olga & Lev Leviev Heart Center The Chaim Sheba Medical Center

Ramat Gan, Tel Hasomer, 5265601, Israel
The University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States
UC San Diego Health - Sulpizio Cardiovascular Center

La Jolla, California, 92037, United States
UOC Cardiologica Azienda Ospedaliera Spedali Civili di Brescia

Brescia, 25123, Italy
UPMC Presbyterian

Pittsburgh, Pennsylvania, 15213, United States
Union Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, 430022, China
Universitaetsklinikum Essen Westdeutsches Herz-Gefaesszentrum

Essen, 45147, Germany
Universitaetsklinikum Heidelberg

Heidelberg, Germany
Universitaetsklinikum Jena Klinik fuer Innere Medizin 1

Jena, 07747, Germany
Universitaetsmedizin Goettingen

Göttingen, 37075, Germany
University Hospital Maastricht

Maastricht, 6229HX, Netherlands
University of California San Francisco

San Francisco, California, 94143, United States
University of Iowa

Iowa City, Iowa, 52242, United States
University of Utah Hospital

Salt Lake City, Utah, 84132, United States
University of Virginia Health System University Hospital

Charlottesville, Virginia, 22908, United States
University of Washington Medical

Seattle, Washington, 98195, United States
Universitätsklinikum Würzburg

Würzburg, Germany
Unniversitaetsklinikum Magdeburg Klinik fur Kardiologie und Anglologie

Magdeburg, 39120, Germany
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States
Westchester Medical Center

Valhalla, New York, 10595, United States
Xiangya Hospital of the Central South University

Changsha, Hunan, 410008, China
Yale School of Medicine

New Haven, Connecticut, 06511, United States
Yale University School of Medicine

New Haven, Connecticut, 06519, United States
Ziv Medical Center

Safed, 13100, Israel

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