A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (age 12 years to < 18 years) with Symptomatic Obstructive Hypertrophic Cardiomyopathy (SCOUT-HCM)

简要总结

主要结局

次要结局

• Change from baseline in the following at Week 28: 1) Resting LVOT gradient 2) Post-exercise peak LVOT gradient 3) Maximal wall thickness 4) Ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e’)
• Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
• Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
• Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
• Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28 Incidence of treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)
• Change from baseline in QT interval on electrocardiogram at Week 28
• Incidence of left ventricular ejection fraction (LVEF) ≤ 30%
• Incidence of LVEF < 50%
• Summary of plasma concentrations by visit (Ctrough and post dose)
• Cmax and AUC using sparse sampling utilizing population PK approach
• 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 HCMSQ SoB domain at Week 28