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
FAPI PET/CT Imaging in Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) (FAPI-ARVC))
Arrhythmogenic right ventricular cardiomyopathy (ARVC) is an inherited heart muscle disease associated with fibrofatty myocardial replacement, ventricular arrhythmias, and an increased risk of sudden cardiac death. Although current diagnostic approaches, including the 2010 Task Force Criteria and the Padua criteria, improve recognition of the disease, early diagnosis remains challenging, particularly when structural abnormalities are subtle or absent on conventional imaging. Echocardiography and cardiac magnetic resonance imaging are central to evaluation, but their sensitivity for early or active fibrotic remodeling may be limited. This limitation may be particularly relevant in patients who are unable to undergo cardiac magnetic resonance imaging, in whom 68Ga-DOTA-SA-FAPI PET/CT may provide complementary diagnostic information. This prospective single-group diagnostic imaging study aims to investigate the incremental value of protocol-specified 68Ga-DOTA-SA-FAPI PET/CT imaging in patients with ARVC. FAPI PET/CT is a novel molecular imaging method that targets activated fibroblasts and may allow non-invasive detection of active myocardial fibrosis.Fifteen adult patients with an established diagnosis of ARVC will undergo protocol-specified 68Ga-DOTA-SA-FAPI PET/CT imaging in addition to clinical evaluation, electrocardiography, echocardiography, and review of previously obtained cardiac magnetic resonance imaging findings. FAPI PET/CT findings will be compared with conventional diagnostic criteria and other clinical and imaging parameters, including previously available cardiac magnetic resonance imaging findings. Participants will also be followed clinically for 6 months after imaging to explore possible associations between FAPI uptake and short-term clinical outcomes, including arrhythmic events, ventricular function, and laboratory markers. The study is expected to provide preliminary evidence on whether 68Ga-DOTA-SA-FAPI PET/CT may improve the detection of myocardial fibrosis and contribute to diagnostic assessment and risk stratification in ARVC. It may also help clarify the potential role of FAPI PET/CT in patients who are unable to undergo cardiac magnetic resonance imaging. The findings may support future larger prospective studies in this field.
[64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy
The primary goal of this pilot study is to determine whether \[64Cu\]FBP8, a novel fibrin-binding positron emission tomography (PET) probe, can identify intracardiac thrombi when paired with simultaneous hybrid cardiac PET/MRI in twenty (20) individuals with transthyretin or light chain cardiac amyloidosis and atrial fibrillation (AF) or atrial flutter (AF). The primary hypothesis of this study is that \[64Cu\]FBP8 PET/MRI can identify intracardiac thrombi in \>90% of subjects with confirmed intracardiac thrombi based on transesophageal echocardiogram (TEE). In secondary analyses, the investigators will seek to determine associations between intracardiac thrombi and left atrial function and left ventricular amyloid burden.
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