A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy

简要总结

主要结局

次要结局

• Change from baseline in resting LVOT gradient(At Week 28)
• Change from baseline in post-exercise peak LVOT gradient(At Week 28)
• Change from baseline in maximal wall thickness(At Week 28)
• Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')(At Week 28)
• Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)(From baseline up to Week 28)
• Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg(From baseline up to Week 28)
• Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28(From baseline up to Week 28)
• Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28(From baseline up to Week 28)
• Number of participants with treatment-emergent adverse events (TEAEs)(Up to Week 218)
• Number of participants with treatment-emergent serious adverse events (TESAEs)(Up to Week 218)
• Change from baseline in electrocardiogram (ECG) (QT interval)(At Week 28)
• Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%(Up to Week 200)
• Number of participants with LVEF < 50%(Up to Week 200)
• Trough observed plasma concentration (Ctrough)(Up to Week 200)
• Post-dose plasma concentration of mavacamten(Up to Week 200)
• Maximum observed concentration (Cmax)(Up to Week 200)
• Area under the concentration-time curve (AUC)(Up to Week 200)
• Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales(At Day 1 and Week 11)
• Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain(At Week 28)