What the trial was testing
The EXPLORER-HCM enrolled 251 patients with hypertrophic cardiomyopathy. The study was sponsored by MyoKardia and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
37% hit the trial's combined goal of better symptoms and exercise — vs. 17% on placebo.
The Lancet · 2020 · NCT03470545
These findings — that of patients hit the combined symptom-and-exercise goal on mavacamten vs. placebo — were published in the The Lancet and represent the headline result of the study.
Researchers tracked outcomes across 251 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with hypertrophic cardiomyopathy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Mavacamten (Camzyos) is FDA-approved for symptomatic obstructive hypertrophic cardiomyopathy and available now. It is a once-daily pill. Heart-pumping function must be checked by ultrasound before and during treatment, and prescribing is restricted to certified providers. Ask a cardiologist who specializes in HCM about eligibility.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open hypertrophic cardiomyopathy trials
Pediatric Cardiomyopathy Mutation Analysis
The goal of this protocol is to obtain information from individuals with cardiomyopathy and from their families in order to elucidate the molecular genetics of this disorder. This will provide the basis for future genetic counseling as well as contribute to elucidating the biology of normal and abnormal cardiac function.
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.