This study aims to evaluate whether, under a treatment strategy primarily guided by left ventricular ejection fraction (LVEF), dose up-titration in adult patients with symptomatic obstructive hypertrophic cardiomyopathy who have received Mavacamten treatment for at least 12 weeks with a stable maintenance dose (as baseline) and with LVEF ≥55% can further reduce left ventricular outflow tract (LVOT) gradient after 24 weeks of treatment. It will also assess changes in hemodynamics, clinical symptoms, cardiac structure, and function. Furthermore, the study will conduct stratified analyses based on patients' baseline resting or Valsalva-provoked LVOT gradient levels at enrollment \[\<30 mmHg vs. ≥30 mmHg\] to explore differences in the aforementioned outcome measures among patients with different gradient levels, with the goal of providing evidence for individualized clinical dose adjustment.
1. Adult patients diagnosed with obstructive hypertrophic cardiomyopathy (HCM) and classified as New York Heart Association (NYHA) functional class II-III. 2. Have received stable Mavacamten treatment for at least 12 weeks with a stable dose. 3. LVEF ≥ 55%.
1. Hypertrophy caused by other systemic diseases with a clear etiology. 2. Planned use of strong CYP2C19 inhibitors, moderate to strong CYP2C19 inducers, or CYP3A4 inducers during the study. 3. Planned use of disopyramide, ranolazine, verapamil combined with beta-blockers, or diltiazem combined with beta-blockers during the study. 4. Previous intolerance to Mavacamten. 5. Severe hepatic or renal dysfunction. 6. Pregnant or lactating women.
Single group
Mavacamten
DrugEligible participants will undergo dose escalation at baseline from their current dose to the next higher level (once daily: 2.5 mg → 5 mg, 5 mg → 10 mg, or 10 mg → 15 mg). At Week 4, clinical status and echocardiography will be re-evaluated, and the same dose will be maintained for the subsequent 8 weeks, unless LVEF is \< 50%. After 12 weeks of treatment at the same dose level: If LVEF remains ≥ 55%, further dose escalation is permitted. If LVEF is between 50% and 55%, the current dose will be maintained. If LVEF is \< 50%, treatment will be interrupted/discontinued.
Change From Baseline in Valsalva Peak Left Ventricular Outflow Tract (LVOT) Pressure Gradient Assessed by Echocardiography at Week 24
Left Ventricular Wall Thickness
Left Atrial Volume Index
Left Ventricular Mass Index
Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)
The Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) includes the physical limitation and total symptom domains. Scores range from 0 to 100, where higher scores indicate better health status, fewer symptoms, and less physical limitation (a better outcome).
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