Hypertrophic cardiomyopathy is a genetic heart disease where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. It affects roughly 1 in 500 people and is the most common cause of sudden cardiac death in young athletes. Treatment traditionally focused on controlling symptoms with beta-blockers, preventing blood clots, and sometimes surgery or alcohol septal ablation to reduce obstruction.
What's actually going on in research
Trials are testing drugs that directly address the underlying muscle thickening and improve the heart's ability to fill and pump. Mavacamten, a cardiac myosin inhibitor approved by the FDA in 2022, reduces obstruction without surgery in many people. Researchers are studying gene therapies for specific mutations, examining the role of small heart proteins called cardiac troponins, and testing whether earlier treatment can prevent progression.
Cardiac myosin inhibitors
These drugs bind to the heart muscle protein myosin and reduce its overactivity, the core problem in hypertrophic cardiomyopathy. Mavacamten is approved, and similar drugs are in trials to see if they work for more forms of the disease.
Gene therapy
Because hypertrophic cardiomyopathy is caused by mutations in genes encoding heart muscle proteins, gene-editing approaches are being tested. Early studies aim to reduce the production of abnormal protein in people with specific mutations.
Troponin modulation
Another class of drugs targets cardiac troponin, a different protein involved in heart muscle contraction. These may offer an alternative for people who don't respond to myosin inhibitors.
What to know before you search
Eligibility often depends on whether there's obstruction in the heart, symptom severity, genetic mutation type, prior treatments, and family history of sudden cardiac death.
What types of trials are currently open
- Myosin inhibitor trials — Testing drugs that reduce heart muscle contraction force and may shrink the thickened heart wall or improve symptoms without surgery.
- Gene therapy trials — Testing one-time treatments that aim to correct or silence the genetic mutation causing the disease in people with specific inherited forms.
- Surgical trials — Comparing newer surgical techniques, alcohol septal ablation, and medication approaches to reduce obstruction and improve quality of life.
- Device trials — Testing implantable defibrillators and pacemakers to prevent sudden cardiac death and improve heart rhythm in high-risk people.
- Natural history studies — Following people with hypertrophic cardiomyopathy over time to understand how the disease progresses and what predicts outcomes.
Recently added Hypertrophic Cardiomyopathy trials
Receive a gene therapy treatment for Friedreich ataxia heart disease
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
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).
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