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
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.