Mavacamtenis a novel, small molecule, selective allosteric inhibitor of cardiac-specific myosin, for the treatment of patients with symptomatic oHCM. This study will assess the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM.
This is a randomized, double-blinded, placebo-controlled clinical study witha long-term extension to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM. Approximately 81eligible participants will be enrolled and randomized in a 2:1 ratio (mavacamten:placebo). Participants will receive mavacamten or matching placebofor 30 weeks indouble-blinded manner. After 30-week double-blinded placebo-controlled treatment, eligible participants will receive mavacamten for additional 48 weeks (placebogroup: switch from placebo to mavacamten, mavacamten group: maintain on mavacamten).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
81
Mavacamten Capsules
Matching PBO capsules during placebo controlled period,and mavacamten capsules during long term extension period
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Change From Baseline to Week 30 in Valsalva Left Ventricular Outflow Tract (LVOT) Peak Gradient
To compare the effect of a 30-week course of mavacamten with placebo on Valsalva LVOT peak gradient as determined by Doppler echocardiography
Time frame: 30 weeks
Change From Baseline to Week 30 in Resting LVOT Peak Gradient
To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction.
Time frame: 30 weeks
Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 30 mmHg at Week 30
To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction.
Time frame: 30 weeks
Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 50 mmHg at Week 30.
To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction.
Time frame: 30 weeks
Proportion of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Classification From Baseline to Week 30
To compare the effect of a 30-week course of mavacamten with placebo on clinical symptoms
Time frame: 30 weeks
Change From Baseline to Week 30 in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS)
To compare the effect of a 30-week course of Mavacamten with placebo on Participant-Reported health status individually The KCCQ (23-item version) is a patient-reported questionnaire that measures the impact of patients' CV disease or its treatment on 6 distinct domains using a 2-week recall: symptoms/signs, physical limitations, quality of life, social limitations, self-efficacy, and symptom stability (Green et al., 2000). In addition to the individual domains, 2 summary scores can be calculated from the KCCQ: the overall summary score (includes the total symptom, physical limitation, social limitations and quality of life scores) and the clinical summary score (combines the total symptom and physical limitation scales). Scores range from 0 to 100, with higher scores reflecting better health status. The KCCQ will be administered to participants as indicated.
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Time frame: 30 weeks
Change From Baseline to Week 30 in N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP)
To compare the effect of a 30-week course of mavacamten on cardiac biomarkers
Time frame: 30 weeks
Change From Baseline to Week 30 in Cardiac Troponin
To compare the effect of a 30-week course of mavacamten on cardiac biomarkers
Time frame: 30 weeks
Change From Baseline to Week 30 in Left Ventricular (LV) Mass Index
To compare the effect of a 30-week course of mavacamten with placebo on LV mass evaluated by cardiac magnetic resonance (CMR) imaging.
Time frame: 30 weeks