Cardiomyopathy is a disease of the heart muscle that makes it harder for the heart to pump blood. It affects about 1 in 500 people and comes in several forms — dilated, hypertrophic, restrictive, and arrhythmogenic. Treatment ranges from medications to slow progression to devices like pacemakers and defibrillators, and in severe cases, heart transplant.
What's actually going on in research
Trials are testing gene therapies for specific inherited forms, SGLT2 inhibitors that showed promise in heart failure, drugs targeting the sarcomere proteins that control heart contraction, and treatments to reduce fibrosis. Researchers are also studying how to identify cardiomyopathy earlier through genetic testing and imaging, before symptoms appear.
Gene therapy
Trials are testing one-time gene therapies for Duchenne muscular dystrophy cardiomyopathy and other genetic forms. Early results suggest these therapies may stabilize or improve heart function in some patients.
Mavacamten and myosin inhibitors
Mavacamten was FDA-approved in 2022 for hypertrophic cardiomyopathy and helps the heart relax. Other myosin inhibitors are now in trials to see if they work for additional forms of the disease.
SGLT2 inhibitors
Drugs like empagliflozin, originally developed for diabetes, have shown benefit in heart failure trials. Studies are testing whether they help specific cardiomyopathy subtypes.
What to know before you search
Eligibility typically depends on cardiomyopathy type, how well the heart is pumping (ejection fraction), genetic mutation if known, and prior treatments including medications and devices.
What types of trials are currently open
- Treatment trials — Testing new medications that target the heart muscle directly, like myosin inhibitors, or repurposed drugs from heart failure studies.
- Gene therapy trials — Testing one-time treatments that deliver corrected genes to heart cells, mainly for inherited forms like Duchenne cardiomyopathy.
- Device trials — Studies of new pacemakers, defibrillators, or ventricular assist devices to support heart function.
- Exercise and rehabilitation trials — Testing whether specific exercise programs are safe and beneficial for people with cardiomyopathy.
- Registry studies — Following large groups of people with cardiomyopathy over time to understand disease progression and treatment outcomes.
Recently added 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 protein-stabilizing therapy while undergoing heart imaging
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
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