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
ENavogliflozin DElivering Alleviation of Ventricular Diastolic Dysfunction in nonObstRuctive Hypertrophic CardioMyopathy
The primary objective of this study is to investigate the effect of enavogliflozin, an Sodium-glucose transporter 2 (SGLT2) inhibitor, compared with placebo on left ventricular diastolic function in patients with nonobstructive hypertrophic cardiomyopathy. The secondary objective of this study is to investigate the effect of enavogliflozin on exercise capacity, symptoms, serum biomarkers, and arrhythmic burden in patients with nonobstructive hypertrophic cardiomyopathy.
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD). Moreover, the specific goals are to generate an alert system for possible cases of RD with data from the electronic medical record, to describe the occurrence of RD in the evaluated population, to characterize the population, to describe patterns of diagnosis and treatment of RD present at the time, and to explore patient-reported outcomes.