This study aims to evaluate the efficacy and safety of Mavacamten combined with radiofrequency ablation compared to Mavacamten alone in patients with symptomatic obstructive hypertrophic cardiomyopathy (HOCM).
Participants were randomized into two groups:
At least 18 years old at screening.
Body weight was greater than 45 kg at screening.
Diagnosed with obstructive hypertrophic cardiomyopathy (oHCM) consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Chinese Society of Cardiology guidelines, ie, satisfy criteria below (criteria to be documented by the echocardiography core laboratory): A. Had unexplained left ventricular (LV) hypertrophy with nondilated ventricular chambers in the absence of other cardiac (eg, hypertension, aortic stenosis) or systemic disease and with maximal LV wall thickness ≥15 mm (or ≥13 mm with positive family history of hypertrophic cardiomyopathy), as determined by core laboratory interpretation, and B. Had left ventricular outflow tract (LVOT) peak gradient ≥50 mm Hg during screening as assessed by echocardiography at rest or after Valsalva maneuver (confirmed by echocardiography core laboratory interpretation).
Had documented LV ejection fraction (LVEF) ≥55% by echocardiography core laboratory read of screening TTE at rest.
1) Participated in a clinical trial in which the participant received any investigational drug (or was currently using an investigational device) within 30 days prior to screening, or at least 5 times the respective elimination half-life (if known), whichever was longer.
Parallel
radiofrequency ablation
Procedure/SurgeryRadiofrequency Ablation is a minimally invasive interventional technique performed via catheter guidance. It utilizes thermal energy (50-80°C) generated by high-frequency alternating current (typically 300-750 kHz) to induce coagulative necrosis or electrophysiological isolation in targeted tissues, thereby eliminating abnormal electrical activity or mechanical obstruction. In the treatment of cardiomyopathy, RFA is applied to ablate hypertrophied myocardial tissue (e.g., the ventricular septum) to alleviate left ventricular outflow tract (LVOT) obstruction.
Mavacamten
DrugInitial Dose: Both groups received a starting dose of Mavacamten 2.5 mg orally once daily. Dose Titration: Adjustments were made based on a previously published titration protocol, guided by correlations among resting left ventricular ejection fraction (LVEF), LVOT gradient during Valsalva maneuver, and pre-dose Mavacamten plasma concentrations.
Change in Valsalva LVOT peak gradient from baseline to week 30, as determined by Doppler echocardiography.
Change in Valsalva LVOT peak gradient from baseline to week 30, as determined by Doppler echocardiography.
The proportion of patients at week 30 with a Valsalva LVOT peak gradient less than 30mmHg.
The proportion of patients at week 30 with a Valsalva LVOT peak gradient less than 30mmHg.
The proportion of patients at week 30 with a Valsalva LVOT peak gradient less than 50 mm Hg.
The proportion of patients at week 30 with a Valsalva LVOT peak gradient less than 50 mm Hg.
The proportion of patients at week 30 at least 1 class improvement in NYHA functional classification.
The proportion of patients at week 30 at least 1 class improvement in NYHA functional classification.
Changes from baseline to week30 in the resting LVOT peak gradient.
Changes from baseline to week30 in the resting LVOT peak gradient
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