Overview
The purpose of this study is to evaluate the safety, tolerability, and efficacy of mavacamten compared with placebo in participants with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
Principal investigator
Eligibility criteria
Inclusion Criteria
* Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines: unexplained left-ventricular hypertrophy with non-dilated ventricular chambers in the absence of other cardiac or systemic disease which can produce the required magnitude of hypertrophy of a maximal left ventricular (LV) wall thickness ≥ 15 millimeters (mm) (or ≥ 13 mm with positive family history of hypertrophic cardiomyopathy \[HCM\]) as determined by core laboratory interpretation.
* Peak left ventricular outflow tract (LVOT) pressure gradient \< 30 millimeters mercury (mm Hg) at rest and \< 50 mm Hg with provocation (Valsalva maneuver and stress echocardiography).
* New York Heart Association (NYHA) Class II or III. Exclusion Criteria
* Known infiltrative or storage disorder causing cardiac hypertrophy that mimics non-obstructive hypertrophic cardiomyopathy (nHCM) such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy.
* History of unexplained syncope within 6 months prior to screening.
* History of sustained ventricular tachyarrhythmia (\> 30 seconds) within 6 months prior to screening.
* Other protocol-defined Inclusion/Exclusion criteria apply.
* Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines: unexplained left-ventricular hypertrophy with non-dilated ventricular chambers in the absence of other cardiac or systemic disease which can produce the required magnitude of hypertrophy of a maximal left ventricular (LV) wall thickness ≥ 15 millimeters (mm) (or ≥ 13 mm with positive family history of hypertrophic cardiomyopathy \[HCM\]) as determined by core laboratory interpretation.
* Peak left ventricular outflow tract (LVOT) pressure gradient \< 30 millimeters mercury (mm Hg) at rest and \< 50 mm Hg with provocation (Valsalva maneuver and stress echocardiography).
* New York Heart Association (NYHA) Class II or III. Exclusion Criteria
* Known infiltrative or storage disorder causing cardiac hypertrophy that mimics non-obstructive hypertrophic cardiomyopathy (nHCM) such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy.
* History of unexplained syncope within 6 months prior to screening.
* History of sustained ventricular tachyarrhythmia (\> 30 seconds) within 6 months prior to screening.
* Other protocol-defined Inclusion/Exclusion criteria apply.
For Referring Providers
Do you have a patient you think would be a good candidate for this trial? Learn more about enrolling your patient.