Cardiomyopathy is a disease of the heart muscle that makes it harder for the heart to pump blood effectively. It includes several distinct types — dilated, hypertrophic, restrictive, and others — each with different causes and treatment needs, from genetic mutations to infections to unknown triggers.
What's actually going on in research
Mavacamten, a cardiac myosin inhibitor, has become the first drug targeting the underlying mechanism of hypertrophic cardiomyopathy and is now being studied in additional subtypes. Gene therapy trials are targeting familial cardiomyopathies caused by mutations in MYBPC3, MYH7, and other sarcomere genes. SGLT2 inhibitors have proven benefits across multiple cardiomyopathy types and are being studied specifically in genetic and inflammatory subtypes.
Cardiac myosin inhibitors
Mavacamten directly reduces the overactive heart muscle contraction in hypertrophic cardiomyopathy. Trials are refining dosing, testing it in obstructive and non-obstructive disease, and exploring other myosin inhibitors.
Gene therapy
For hereditary cardiomyopathies caused by single-gene mutations, early-phase gene therapy trials are delivering corrected genes via viral vectors directly to heart muscle cells.
SGLT2 inhibitors in HCM
These diabetes drugs have shown broad heart benefits and are now being specifically tested in hypertrophic and other cardiomyopathies to reduce symptoms and disease progression.
What to know before you search
Eligibility depends on cardiomyopathy type, genetic mutation identified, LVOT gradient (for HCM), ejection fraction, and symptom severity.
What types of trials are currently open
- Drug trials — Testing new cardiac medications, cardiac myosin inhibitors, or SGLT2 inhibitors in specific cardiomyopathy types.
- Gene therapy trials — Evaluating gene correction approaches for hereditary cardiomyopathy caused by specific mutations.
- Device trials — Testing implantable defibrillators, cardiac resynchronization therapy, and mechanical assist devices.
- Surgical trials — Comparing septal reduction procedures for obstructive hypertrophic cardiomyopathy.
- Observational studies — Tracking genotype-phenotype relationships and natural history in inherited cardiomyopathies.
Recently added Cardiomyopathy trials
Prospective Investigation of Cirrhotic Cardiomyopathy in Humans
Cirrhotic Cardiomyopathy (CCM) is a recognized complication of cirrhosis, but understudied despite recent retrospective data suggesting it may be common, affecting one in three patients with decompensated cirrhosis, and associated with significantly increased risk of death and adverse hepatic and cardiac events. Moreover, evidence from preclinical models and children suggest elevated bile acids in the blood may contribute to CCM, but data from adults with cirrhosis are scarce. Therefore, we are conducting the first contemporary prospective multi-center investigation of CCM in adults in the USA to define CCM risk factors and impact on outcomes while deepening understanding of the role of bile acids in development of this disease.
ATROPOS: Survival Analysis of ICI-associated Myocarditis
ATROPOS is an international, registry-based observational study of cancer patients with immune checkpoint inhibitor-associated myocarditis (ICI-M). The study evaluates whether immunosuppressive treatment strategies, including first-line glucocorticoids and subsequent second-line immunosuppressants, are associated with overall mortality, and whether these associations vary according to myocarditis severity. The target number of cases to be included is 1500 spanning from at least 160 centers, located in at least 18 countries. Analyses will adjust for clinically relevant confounders and may use time-dependent survival models where appropriate.
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