Cardiomyopathy describes several diseases where the heart muscle weakens, stiffens, or thickens, making it harder to pump blood. The most common types are dilated (enlarged heart), hypertrophic (thickened walls), and restrictive (stiff heart). Treatment ranges from medications and devices to heart transplant in severe cases.
What's actually going on in research
Trials are testing gene therapies for inherited forms, drugs that target the thick heart muscle in hypertrophic cardiomyopathy, and medications that address the underlying protein misfolding in certain genetic types. Researchers are also studying stem cells, cardiac MRI to predict who needs treatment, and ways to prevent progression in people with early disease.
Gene therapy
Several trials are testing one-time gene treatments for inherited cardiomyopathies caused by single gene mutations. Early results show some people's heart function improved after receiving the therapy.
Mavacamten and cardiac myosin inhibitors
Mavacamten, approved by FDA in 2022, reduces the thick heart muscle in hypertrophic cardiomyopathy by blocking overactive myosin proteins. Newer drugs in this class are being tested to see if they work faster or reach more patients.
Protein stabilizers
Drugs like tafamidis stabilize misfolded proteins in certain inherited cardiomyopathies, slowing heart damage. Trials are testing similar approaches for other genetic forms where proteins clump in the heart.
What to know before you search
Eligibility typically depends on cardiomyopathy type, heart function measures, genetic test results if inherited, symptoms, and prior treatments including medications and devices.
What types of trials are currently open
- Treatment trials — Testing new medications to improve heart function, reduce symptoms like shortness of breath, or slow disease progression.
- Gene therapy trials — Testing one-time treatments that deliver corrected genes to heart muscle cells in people with inherited cardiomyopathy.
- Device trials — Testing implantable devices like pacemakers or defibrillators that help the heart beat more effectively or prevent sudden cardiac arrest.
- Imaging studies — Using advanced MRI or ultrasound to identify early disease and predict who will benefit from treatment.
- Registry studies — Following large groups of people with cardiomyopathy over time to learn what factors predict outcomes and treatment response.
Recently added Cardiomyopathy trials
Complete advanced heart imaging scans to map cardiac energy patterns
The study is designed to establish a comprehensive myocardial energetic phenotype across a broad spectrum of cardiovascular diseases using phosphorus-31 magnetic resonance spectroscopy (31P-MRS). And the study plans to enroll 1,000 participants, comprising healthy volunteers and patients with diverse cardiac conditions. Target cohorts primarily encompass heart failure syndromes and various cardiomyopathies, with a particular focus on non-ischemic and hypertrophic phenotypes, as well as ischemic and other structural etiologies. By integrating 31-MRS with multiparametric standard CMR, this study aims to map disease-specific metabolic signatures. The primary outcome measure is the myocardial PCr/ATP ratio, serving as a direct in vivo marker of energetic impairment. Furthermore, the study aims to explore the coupling between metabolic phenotypes and structural remodeling (tissue characterization), mechanical dysfunction, and systemic clinical biomarkers. Crucially, the study will evaluate the prognostic value of these energetic phenotypes in predicting clinical outcomes, ultimately providing novel insights into the pathophysiology and risk stratification of cardiomyopathies and heart failure.
Differentiating Heart Disease Between Adult-onset Autoimmune and Type 2 Diabetes
Background: The study of factors associated with diabetic heart disease (DHD), the leading cause of mortality in diabetes, represents an unmet need. Autoimmune diabetes in adults (AID) and type 2 diabetes (T2D) differ in pathophysiology, vascular complications, and autonomic impairment, with significant implications for DHD, which results from a complex interplay among coronary micro- and macro-vascular circulation, neuro-immune control, and myocardial damage. Objective: To characterize the complexity of DHD and its determinants by comparing AID and T2D. Methods: DHD will be studied in individuals with AID, T2D, and healthy controls matched for cardiovascular risk factors. Advanced echocardiography and cardiovascular magnetic resonance will allow high-quality assessment of the different components of DHD (such as cardiac geometry, performance, and contraction). To identify the determinants of DHD, cardiac study results will be correlated with autonomic dysfunction tests, markers of small fiber neuropathy, and laboratory assessments of metabolic alterations, cardiac autoimmunity, and the inflammasome. Furthermore, genetic susceptibility to autoimmune diabetes and pancreatic autoimmunity will also be evaluated and correlated with DHD
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