Dilated cardiomyopathy means the heart's main pumping chamber stretches and weakens, making it harder to pump blood. It affects about 1 in 2,500 people and can be inherited or caused by infections, alcohol, chemotherapy, or unknown factors. Treatment includes medications to reduce strain on the heart, devices like pacemakers or defibrillators, and sometimes heart transplant.
What's actually going on in research
Trials are testing gene therapies for inherited forms, SGLT2 inhibitors that protect the heart, RNA-targeting drugs for specific genetic mutations, and stem cell approaches to repair heart muscle. Researchers are also studying how to identify people at risk before symptoms appear, and testing drugs that address the underlying causes rather than just managing symptoms.
Gene therapy
Trials are testing whether delivering healthy gene copies can correct inherited dilated cardiomyopathy caused by mutations in genes like MYBPC3 and LMNA. Early results suggest some people's heart function improves after a single treatment.
SGLT2 inhibitors
Drugs originally developed for diabetes, including dapagliflozin and empagliflozin, have been shown to reduce hospitalizations and death in people with heart failure. Trials are testing whether they help specifically in dilated cardiomyopathy.
RNA-silencing drugs
For people with mutations in the TTR gene that cause heart muscle stiffening, drugs like patisiran can silence the faulty gene. Researchers are exploring whether similar approaches can work for other genetic forms of dilated cardiomyopathy.
What to know before you search
Eligibility typically depends on how weak the heart's pumping function is, genetic test results, symptoms like shortness of breath, and prior treatments including medications and devices.
What types of trials are currently open
- Drug trials — Testing new heart failure medications, including drugs that improve pumping strength or reduce strain on the heart muscle.
- Gene therapy trials — Testing one-time treatments that deliver corrected gene copies to heart muscle cells in people with inherited dilated cardiomyopathy.
- Device trials — Testing new pacemakers, defibrillators, or devices that help the heart pump more effectively.
- Stem cell trials — Testing whether injecting stem cells into the heart can repair damaged muscle and improve pumping function.
- Natural history studies — Following people with dilated cardiomyopathy over time to understand how the disease progresses and what predicts outcomes.
Recently added Dilated Cardiomyopathy trials
Take a daily pill to evaluate long-term effects on inherited heart disease
Danicamtiv is an investigational medication, which means it is still being studied and has not been approved. The purpose of this study is to learn how well danicamtiv works, how safe it is, how well people can take it when used in patients with genetic or familial dilated cardiomyopathy (DCM). There are currently no approved medicines made specifically to treat genetic or familial DCM.
Take a diabetes medicine being tested for pediatric heart failure
the study to investigate the efficacy of empagiflozin in pediatric heart failure
Find Dilated Cardiomyopathy trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.