Obstructive Hypertrophic Cardiomyopathy
Conditions
Interventions
Control group:This arm receives oral mavacamten, which is titrated to the maximum tolerated dose and is subsequently maintained at a stable dosage.
Sponsors
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1. Aged 18 to 80 years, regardless of gender. 2. Maximal ventricular septal wall thickness >=15 mm, or >=13 mm in participants with a family history of oHCM. 3. Resting or Valsalva-provoked left ventricular outflow tract peak gradient >=50 mm Hg. 4. Refractory to or intolerant of beta-blockers and/or non-dihydropyridine calcium channel blockers. 5. New York Heart Association (NYHA) class III or NYHA class II with the following conditions: age = 100 mmHg; or experiencing mitral regurgitation >=3+ caused by systolic anterior motion (SAM) of the mitral valve during a resting state. 6. Informed of the nature of the clinical trial, consented to participate in all of the activities, and signed an informed consent form.
Exclusion criteria
Exclusion criteria: 1. Administration of mavacamten (CAMZYOS®) within 30 days prior to screening. 2. Presented with concomitant diseases, such as native valvular disease, coronary artery disease or myocardial bridge, which needed open-heart surgery. 3. Left ventricular ejection fraction 500 msec, or any other electrocardiogram abnormality considered by the investigator to pose a potential risk to the subject's safety. 14. Patients who are unable to cooperate with screening examinations due to specific reasons, such as failure to undergo cardiac magnetic resonance imaging due to atrial fibrillation, or due to an implantable cardioverter-defibrillator or pacemaker; or inability to adequately perform cardiopulmonary exercise testing (RER < 1.0) or Valsalva maneuver. 15. Known infiltrative or storage diseases that can lead to cardiac hypertrophy resembling hypertrophic obstructive cardiomyopathy, such as Fabry disease, amyloidosis, or Noonan syndrome with associated left ventricular hypertrophy. 16. Patients diagnosed with malignant tumors. 17. Known to be allergic to heparin, contrast agents, or protamine. 18. Known to be allergic to mavacamten. 19. Current use or use within 14 days prior to screening of prohibited medications, including cytochrome P450 (CYP) 2C19 inhibitors (e.g., omeprazole or esomeprazole), strong CYP3A4 inhibitors (e.g., itraconazole), or St. John’s wort (Hypericum perforatum). 20. Participating in clinical trials of other drugs or medical devices without achieving the primary endpoint or completing the primary endpoint observation for less than 3 months. 21. Presence of any other condition deemed by the investigator to render the patient unsuitable for trial participation, such as severe hepatic or renal dysfunction, coagulopathy, History of drug use and/or mental disorders, or uncontrolled active infection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| All-cause death;Meet the target left ventricular outflow tract peak gradient threshold;Increase in Peak VO2 by >1.5 mL/kg/min;Mitral regurgitation =10 points;Improvement in NYHA class by >=1 class; | — |
Secondary
| Measure | Time frame |
|---|---|
| Peak oxygen consumption, Peak VO2;Minute ventilation/CO2 production slope, VE/VCO2 slope;Peak respiratory exchange ratio, Peak RER;Resting left ventricular outflow tract gradient, R-LVOTG;Valsalva left ventricular outflow tract gradient, V-LVOTG;Post-exercise LVOT peak gradient, E-LVOTG;Mitral regurgitation;Systolic anterior motion;Left ventricular outflow tract diameter;End-diastolic interventricular septal thickness;Left ventricular ejection fraction;Left atrial volume index;The ratio of early diastolic mitral inflow velocity to early diastolic mitral annular tissue velocity (E/e');The ratio of early diastolic left ventricular filling velocity to atrial contraction–mediated filling velocity (E/A);Maximum tricuspid regurgitation velocity;Left ventricular mass index;N-terminal pro-B-type natriuretic peptide or Brain Natriuretic Peptide (NT-proBNP);cardiac Troponin I/high-sensitivity cardiac Troponin I/cardiac Troponin T (cTnI/hs-cTnI/cTnT);Heart function of New York Heart Association Function (NYHA);The distance of 6-minute walking distance;Kansas City Cardiomyopathy Questionnaire (KCCQ) score;Hypertrophic cardiomyopathy symptom questionnaire (HCMSQ) score;New-onset atrial fibrillation;Meet the guideline-recommended criteria for septal reduction therapy; | — |
Countries
China
Contacts
Public ContactXiang Wei
Tongji Hospital, Tongji Medical CollegeHuazhong University of Science and Technology
Outcome results
None listed