Hypertrophic cardiomyopathy (HCM) is a genetic heart muscle disease in which the left ventricle becomes abnormally thick, impairing its ability to fill and sometimes causing outflow obstruction, heart failure, arrhythmias, or sudden cardiac death. It is one of the most common inherited heart conditions, affecting roughly 1 in 500 people, and most carriers live normal lives though a subset experience serious complications.
What's actually going on in research
Mavacamten, a cardiac myosin inhibitor, became the first drug specifically targeting the underlying mechanism of HCM and is now approved for obstructive HCM with heart failure symptoms. A second myosin inhibitor, aficamten, is in late-stage trials. Beyond obstruction relief, researchers are studying rhythm management, genetic screening strategies, and whether early treatment in gene-positive but symptom-free individuals can prevent disease progression.
Cardiac myosin inhibitors
Mavacamten is now approved for symptomatic obstructive HCM, and aficamten is in late-stage trials; both drugs reduce obstruction and symptoms by directly targeting the overactive cardiac muscle machinery.
Early preventive treatment
Trials are examining whether starting myosin inhibitors in gene-positive individuals who have structural changes but no symptoms can delay or prevent the development of significant hypertrophy.
Arrhythmia management
Atrial fibrillation is common in HCM and worsens outcomes; trials are studying optimal anticoagulation strategies, rhythm control drugs, and catheter ablation approaches tailored to HCM patients.
What to know before you search
Eligibility depends on obstructive versus non-obstructive HCM, symptom severity, left ventricular outflow gradient, and prior treatments.
What types of trials are currently open
- Myosin inhibitor trials — Testing cardiac myosin-blocking drugs to relieve obstruction and reduce symptoms in obstructive HCM.
- Early intervention trials — Evaluating treatment of gene carriers before significant symptoms develop.
- Arrhythmia trials — Studying rhythm control and anticoagulation strategies for HCM-related atrial fibrillation.
- Sudden death prevention trials — Refining risk scores and defibrillator indications to identify who benefits most from implantable devices.
- Genetic screening trials — Testing cascade screening programs for relatives of affected individuals.
Recently added Hypertrophic Cardiomyopathy trials
Mavacamten Outcomes in Hypertrophic Cardiomyopathy (HCM) and the Associated Patient and Physician Experiences in the US
The purpose of this study is to understand the real-world clinical outcomes and treatment patterns of adults with obstructive hypertrophic cardiomyopathy (HCM) treated with mavacamten, and to understand patient and physician experiences with mavacamten treatment, in the US community-based practice
Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan (MANAGE-HCM)
The purpose of this study is to assess the real-world effectiveness and safety of mavacamten in adults diagnosed with symptomatic obstructive hypertrophic cardiomyopathy (HOCM) receiving cibenzoline in Japan
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