Hypertrophic cardiomyopathy (HCM) is a genetic heart condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. It affects about 1 in 500 people and is the most common cause of sudden cardiac death in young athletes. Treatment traditionally focused on managing symptoms with beta-blockers and preventing dangerous heart rhythms, but newer approaches now target the underlying disease mechanism.
What's actually going on in research
Trials are testing cardiac myosin inhibitors that directly reduce the heart's excessive contraction force, gene therapies aimed at correcting the underlying mutations, and drugs to prevent the progression from early gene carrier status to full disease. Researchers are also studying better ways to predict who needs an implanted defibrillator and how to prevent the heart muscle from becoming scarred and stiff over time.
Cardiac myosin inhibitors
Mavacamten, FDA-approved in 2022, was the first drug to directly target the heart's overactive contraction. Additional myosin inhibitors are now in trials, aiming to improve symptoms and potentially reduce the need for septal reduction surgery.
Gene-targeted therapies
Several trials are testing ways to silence the mutated genes that cause HCM, using approaches like antisense oligonucleotides. The goal is to prevent disease progression in people who carry the mutation but haven't yet developed symptoms.
Fibrosis prevention
Researchers are studying drugs that might prevent or reverse the scarring and stiffness that develop in the heart muscle over time. This could address the progressive heart failure that affects some people with HCM.
What to know before you search
Eligibility typically depends on genetic testing results, degree of heart muscle thickening, symptom severity, left ventricular outflow obstruction, and whether you've had prior treatments like septal reduction procedures.
What types of trials are currently open
- Treatment trials — Testing new cardiac myosin inhibitors and other drugs that reduce symptoms like shortness of breath and chest pain, often comparing them to standard beta-blockers or current therapies.
- Disease-modifying trials — Testing whether drugs can slow or prevent the heart muscle from thickening further, measured by echocardiogram and cardiac MRI over time.
- Gene therapy trials — Testing treatments that aim to silence or correct the genetic mutations causing HCM, particularly in people with early or pre-symptomatic disease.
- Prevention trials — Studies in people who carry HCM gene mutations but haven't developed symptoms yet, testing whether early treatment can prevent the disease from appearing.
- Registry studies — Long-term tracking of people with HCM to understand disease progression, identify risk factors for sudden death, and determine who benefits most from defibrillators.
Recently added Hypertrophic Cardiomyopathy trials
Take increasing doses of an experimental heart therapy being tested for safety
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
Take a new heart medication being tested against metoprolol
The main purpose of this trial is to find out whether BHB-1893 works better than metoprolol in adults with symptoms of obstructive hypertrophic cardiomyopathy (oHCM). The trial includes adults who are currently being treated for oHCM or who have not yet been treated.
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