What type of study is this?

This is a Human Clinical Trial study (also classified as: RCT).

What question did this study set out to answer?

The aim is to evaluate the efficacy and safety of mavacamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy.

January 9, 2026Heart Failure Reviews

Evaluating the role of mavacamten in symptomatic non-obstructive hypertrophic cardiomyopathy: the ODYSSEY-HCM trial

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Key result

Mavacamten did not significantly improve peak oxygen uptake or Kansas City Cardiomyopathy Questionnaire scores in symptomatic non-obstructive hypertrophic cardiomyopathy after 48 weeks.

Authors

KHKaveh HosseiniUniversity of Copenhagen SKSina Kazemian SHSivadasanpillai HarikrishnanSree Chitra Thirunal Institute for Medical Sciences and Technology

Discussion

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Implication

Phase 3 trial evaluates mavacamten's impact on functional capacity in symptomatic non-obstructive hypertrophic cardiomyopathy, suggesting the need for further research.

Key Points

The aim is to evaluate the efficacy and safety of mavacamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy.
Phase 3, randomized, double-blind, placebo-controlled trial
Enrolled adults with symptomatic non-obstructive hypertrophic cardiomyopathy and ejection fraction ≥ 60%
Patients received mavacamten or placebo for 48 weeks
Dose titration guided by serial echocardiography
Co-primary endpoints included peak oxygen uptake and Kansas City Cardiomyopathy Questionnaire score
Mavacamten showed pharmacodynamic effects with reductions in myocardial stress markers
No statistically significant improvement in peak oxygen uptake or clinical summary score at 48 weeks
Increased incidence of reversible decreases in ejection fraction requiring dose interruption
Target engagement demonstrated but no definitive patient-centered benefits observed

Cite This Study

Hosseini et al. (2025) studied this question. Mavacamten did not significantly improve peak oxygen uptake or Kansas City Cardiomyopathy Questionnaire scores in symptomatic non-obstructive hypertrophic cardiomyopathy after 48 weeks.

synapsesocial.com/papers/696135ac766f0a95ea6ade57 https://doi.org/10.1007/s10741-025-10578-z

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Implication

Phase 3 trial evaluates mavacamten's impact on functional capacity in symptomatic non-obstructive hypertrophic cardiomyopathy, suggesting the need for further research.