Hypertrophic cardiomyopathy
Conditions
Brief summary
Incidence of major adverse cardiac events (death, stroke, acute myocardial infarction), Incidence of hospitalizations (both cardiovascular [CV] and non-CV), Incidence of heart failure (HF) events (includes HF hospitalizations and urgent emergency room/outpatient visits for HF), Incidence of atrial fibrillation/flutter (new from Screening Visit), Incidence of ICD discharges and resuscitated cardiac arrest, Incidence of ventricular tachyarrhythmias (includes ventricular tachycardia or ventricular fibrillation), Incidence of any AE potentially linked to QT prolongation (Torsade de pointes, CV or sudden death, sustained ventricular tachycardia, ventricular fibrillation and flutter, syncope, and seizures), Frequency and severity of treatment-emergent AEs, treatment-emergent serious AEs, and laboratory abnormalities (including trends in NT-proBNP), For Participants from EXPLORER-HCM Change from Week 24 in left ventricular (LV) mass index For Participants from MAVERICK-HCM Change from baseline in LV mass index
Detailed description
Change from baseline in ECHO parameters of systolic function (eg, LVEF) and diastolic function (eg, peak velocity of early diastolic septal and lateral mitral annular motion [eꞌ], ratio of peak velocity of early diastolic transmitral flow [E] to eꞌ [E/eꞌ], ratio of E to peak velocity of late transmitral flow [A] [E/A], pulmonary artery systolic pressure, or left atrium size) over time., Change from baseline in resting and post-Valsalva LVOT gradient (EXPLORER-HCM participants only), Change from baseline in New York Heart Association functional class over time, Change from baseline in NT-proBNP over time, Frequency of cardiac transplantation
Interventions
DRUGMavacamten
Sponsors
Myokardia Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of major adverse cardiac events (death, stroke, acute myocardial infarction), Incidence of hospitalizations (both cardiovascular [CV] and non-CV), Incidence of heart failure (HF) events (includes HF hospitalizations and urgent emergency room/outpatient visits for HF), Incidence of atrial fibrillation/flutter (new from Screening Visit), Incidence of ICD discharges and resuscitated cardiac arrest, Incidence of ventricular tachyarrhythmias (includes ventricular tachycardia or ventricular fibrillation), Incidence of any AE potentially linked to QT prolongation (Torsade de pointes, CV or sudden death, sustained ventricular tachycardia, ventricular fibrillation and flutter, syncope, and seizures), Frequency and severity of treatment-emergent AEs, treatment-emergent serious AEs, and laboratory abnormalities (including trends in NT-proBNP), For Participants from EXPLORER-HCM Change from Week 24 in left ventricular (LV) mass index For Parti | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in ECHO parameters of systolic function (eg, LVEF) and diastolic function (eg, peak velocity of early diastolic septal and lateral mitral annular motion [eꞌ], ratio of peak velocity of early diastolic transmitral flow [E] to eꞌ [E/eꞌ], ratio of E to peak velocity of late transmitral flow [A] [E/A], pulmonary artery systolic pressure, or left atrium size) over time., Change from baseline in resting and post-Valsalva LVOT gradient (EXPLORER-HCM participants only), Change from baseline in New York Heart Association functional class over time, Change from baseline in NT-proBNP over time, Frequency of cardiac transplantation | — |
Countries
Belgium, Czechia, Denmark, France, Germany, Italy, Netherlands, Poland, Portugal, Spain
Outcome results
None listed