The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
44
Specified dose on specified days
Specified dose on specified days
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
Time frame: At Week 28
Change from baseline in resting LVOT gradient
Time frame: At Week 28
Change from baseline in post-exercise peak LVOT gradient
Time frame: At Week 28
Change from baseline in maximal wall thickness
Time frame: At Week 28
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
Time frame: At Week 28
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Time frame: From baseline up to Week 28
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Time frame: 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
Time frame: From baseline up to Week 28
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
Time frame: From baseline up to Week 28
Number of participants with treatment-emergent adverse events (TEAEs)
Time frame: Up to Week 218
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Local Institution - 0017
Birmingham, Alabama, United States
Local Institution - 0050
Phoenix, Arizona, United States
Local Institution - 0032
Los Angeles, California, United States
Local Institution - 0008
Los Angeles, California, United States
Local Institution - 0033
Palo Alto, California, United States
Local Institution - 0044
San Diego, California, United States
Local Institution - 0038
Aurora, Colorado, United States
Local Institution - 0031
St. Petersburg, Florida, United States
Local Institution - 0053
Atlanta, Georgia, United States
Local Institution - 0013
Chicago, Illinois, United States
...and 37 more locations
Number of participants with treatment-emergent serious adverse events (TESAEs)
Time frame: Up to Week 218
Change from baseline in electrocardiogram (ECG) (QT interval)
Time frame: At Week 28
Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%
Time frame: Up to Week 200
Number of participants with LVEF < 50%
Time frame: Up to Week 200
Trough observed plasma concentration (Ctrough)
Time frame: Up to Week 200
Post-dose plasma concentration of mavacamten
Time frame: Up to Week 200
Maximum observed concentration (Cmax)
Time frame: Up to Week 200
Area under the concentration-time curve (AUC)
Time frame: Up to Week 200
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
Time frame: At Day 1 and Week 11
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
Time frame: At Week 28