Familial Hypertrophic Cardiomyopathy (HCM) Clinical Trials

Familial hypertrophic cardiomyopathy (HCM) is an autosomal dominant genetic heart disease characterized by unexplained left ventricular hypertrophy caused by over 1,000 pathogenic mutations identified in genes encoding sarcomeric proteins. The most commonly affected genes include MYH7 (encoding cardiac myosin heavy chain), MYBPC3 (encoding cardiac myosin-binding protein C), and TNNT2 (encoding cardiac troponin T). The hypertrophy is typically asymmetric with preferential involvement of the anterior ventricular septum, frequently leading to left ventricular outflow tract (LVOT) obstruction in approximately 25% of patients.

The most common symptoms of Familial Hypertrophic Cardiomyopathy include Chest pain and dyspnea on exertion, Syncope or presyncope, Palpitations and arrhythmias, Fatigue and exercise intolerance, Orthopnea in advanced disease, among others. Familial Hypertrophic Cardiomyopathy affects approximately 1 in 500 people; 50% of first-degree relatives of affected individuals inherit the mutation. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Familial Hypertrophic Cardiomyopathy from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Two cardiac myosin inhibitors are FDA approved for symptomatic obstructive hypertrophic cardiomyopathy: mavacamten (Camzyos), approved for adults in April 2022 and for adolescents aged 12 to 17 on September 30, 2026, and aficamten (Myqorzo), approved for adults in December 2025. Both require echocardiograms before and during treatment and are available only through restricted safety programs (REMS) because they can weaken the heart's pumping and cause heart failure.

Yes. As of September 2026, Trial Friend lists 2 FDA-approved drugs used to treat Familial Hypertrophic Cardiomyopathy: Camzyos (mavacamten), first approved in 2022; and Myqorzo (aficamten), first approved in 2025.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Familial Hypertrophic Cardiomyopathy is caused by variants in any of many genes, most often MYBPC3, MYH7, TNNT2, TNNI3, TPM1 and MYL2. MYBPC3 and MYH7 are the most common causes, but about 70% of nonsyndromic HCM has no identifiable variant in the known sarcomere genes.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. The condition affects approximately 1 in 500 people; 50% of first-degree relatives of affected individuals inherit the mutation. A multigene panel identifies the cause in most families.

Yes. Familial Hypertrophic Cardiomyopathy can be caused by variants in many different genes (MYBPC3, MYH7, TNNT2, TNNI3, TPM1 and MYL2 are among the most common), so testing is usually a multigene panel or exome sequencing rather than one gene at a time. MYBPC3 and MYH7 are the most common causes, but about 70% of nonsyndromic HCM has no identifiable variant in the known sarcomere genes.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

Knowing the exact gene matters: it decides which trials a patient qualifies for and which relatives should be tested. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Familial Hypertrophic Cardiomyopathy clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Familial Hypertrophic Cardiomyopathy clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.