symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM)
Conditions
Interventions
Mavacamten Arm: Mavacamten Participants will receive treatment with 1, 2.5, 5, 10, 15 mg dose once daily.
Placebo Arm: Placebo Participants will receive treatment once daily.
Sponsors
Aronson Ron
Eligibility
Sex/Gender
All
Inclusion 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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| -Change from baseline in Kansas City Cardiomyopathy Questionnaire (23-item) Clinical Summary Score (KCCQ-23 CSS) at Week 48 [ Time Frame: Up to Week 48 ] -Change from baseline in peak oxygen consumption (pVO2) at Week 48 [ Time Frame: Up to Week 48 ] | — |
Secondary
| Measure | Time frame |
|---|---|
| -Change from baseline in ventilatory efficiency (VE/VCO2) slope to Week 48 [ Time Frame: Up to Week 48 ] -Proportion of participants with at least 1 class of New York Heart Association (NYHA) improvement from baseline to Week 48 [ Time Frame: Up to Week 48 ] -Change from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) to Week 52 [ Time Frame: Up to Week 48 ] -Change from baseline in cardiac troponin-T (cTn-T) to Week 48 [ Time Frame: Up to Week 48 ] -Change from baseline in hypertrophic cardiomyopathy symptom questionnaire-shortness of breath (HCMSQ-SoB) domain to Week 48 [ Time Frame: Up to Week 48 ] -Time to first major adverse cardiovascular events (MACE)-plus events defined as any cardiovascular (CV) death, nonfatal myocardial infarction, non-fatal stroke, hospitalization for heart failure, or hospitalization for arrhythmias [ Time Frame: Up to 120 Weeks ] | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, Czech Republic, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Korea, Netherlands, Norway, Poland, Portugal, Spain, United Kingdom, United States
Contacts
Public ContactRon Aronson
Bristol-Myers Squibb
Outcome results
None listed