What the trial was testing
The SEQUOIA-HCM enrolled 282 patients with hypertrophic cardiomyopathy. The study was sponsored by Cytokinetics 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
1.7 mL/kg/min greater rise in peak oxygen uptake — a real exercise gain.
New England Journal of Medicine · 2024 · NCT05186818
These findings — that greater improvement in peak exercise oxygen uptake on aficamten over 24 weeks — were published in the New England Journal of Medicine and represent the headline result of the study.
Researchers tracked outcomes across 282 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
Aficamten is under FDA review and not yet approved as of early 2026. A related drug, mavacamten (Camzyos), is FDA-approved for the same condition and available now. Ask a cardiologist who specializes in hypertrophic cardiomyopathy about approved options or whether you qualify for an aficamten trial.
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.