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
Take a new heart medication after switching from another
This is an investigator-initiated two-center study. The goal of this study is to investigate the feasibility, safety and efficacy outcomes of a seamless transition from mavacamten to aficamten in patients with obstructive hypertrophic cardiomyopathy (oHCM).
Try a guided exercise program designed for your heart condition
Hypertrophic Cardiomyopathy (HCM) is an inherited cardiac condition that affects the heart muscle of adults and children. Historically, doctors have been cautious about encouraging patients with HCM to exercise, as they were worried it could be unsafe due to the risk of heart problems or emergencies. But recent evidence shows that the risks of exercise for a patient with HCM are far lower than previously thought and exercise has many benefits for a child's general health and mental well-being and is an important part of their social development. One way to help children with HCM feel confident to exercise is with an individualised exercise 'prescription'. This is where a team of specialist doctors, nurses, and physiotherapists assess patients and advise them on how to exercise safely and effectively. They then provide an exercise plan or 'prescription' individualised to them. This study aims to establish whether the investigators can deliver an individualised exercise prescription (IEP) service for children with HCM who attend outpatient cardiology. A further aim is to see if children will want to take part and adhere to the IEP. In this study children aged 10 to 18 years old attending outpatient cardiology clinic at Alder Hey Children's Hospital (AHCH) under screening for HCM will be asked if they wish to take part in the study. Those who wish to take part will have extra appointments at AHCH, during which the investigators will measure their height and weight. They will also have other tests done like blood pressure, a cycle test, sit-to-stand test, measurement of their hamstring flexibility and an ultrasound measure of their thigh muscle. The participants will be asked to complete a questionnaire about their quality of life. The participants will then be given an individualised exercise plan. In the first month, they will be given a supervised exercise session and asked to record their activity levels in an exercise log. The participants will have either a monthly telephone call or a face-to-face visit to see how they are getting on. After 6 months, all the initial tests will be repeated. The investigators will look at the data to see if the exercise prescription was helpful, if children could stick to it and if there have been improvements in their fitness and quality of life. Finally, the investigators will see them again six months later to see if the IEP programme has had a lasting impact on their exercise levels, fitness, and well-being.
Find Hypertrophic Cardiomyopathy trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.