The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Period 1: Treatment Period
Males and females between 12 and < 18 years of age at screening and at Day 1.
Body weight ≥ 45 kg for the initial cohort and then body weight ≥ 35 kg after at least 10 participants in the initial cohort have undergone dose titration up to Week 4 without observed events of LVEF < 50% at the starting dose of 5 mg qd.
Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other cardiac disease.
Period 1: Treatment Period
Significant valvular heart disease.
Moderate or severe valvular aortic stenosis or fixed subaortic obstruction.
Mitral regurgitation that is greater than mild in severity and not due to systolic anterior motion of the mitral valve (per judgment of Principal Investigator or designee).
Parallel
Aficamten
DrugOral Tablet
Placebo
DrugOral Tablet
Change from baseline in Valsalva left ventricular outflow tract gradient (LVOT-G)
Change from baseline in resting LVOT-G
Change in values of NT-proBNP
Change in values of hs-cTnI
Change in New York Heart Association (NYHA) Functional Class
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