A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy( HORIZON-HCM )
- Conditions
- Obstructive Hypertrophic Cardiomyopathy
- Registration Number
- JPRN-jRCT2031220178
- Lead Sponsor
- Florea Victoria
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 30
Age 18 and greater, body weight >- 35kg
- Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
- Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation
Society guidelines
- Has documented left ventricular ejection fraction (LVEF) >-60% NYHA Class II or III
- Known infiltrative or storage disorder causing cardiac hypertrophy that
mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome
with LV hypertrophy
- History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
- History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for lifethreatening ventricular arrhythmia within 6 months prior to Screening
- Paroxysmal atrial fibrillation with atrial fibrillation present at the time of Screening.
- Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
- Treatment (within 14 days prior to Screening) or planned treatment during
the study with cibenzoline, disopyramide or ranolazine
- Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
- Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the
study
- ICD placement within 2 months prior to Screening or planned ICD placement during the study
- Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation procedures, or completion
- Prior treatment with cardiotoxic agents such as doxorubicin or similar
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change from Baseline to Week 30 in post exercise left ventricular outflow tract (LVOT) peak gradient as determined by Doppler echocardiography
- Secondary Outcome Measures
Name Time Method 1.Change from Baseline to Week 30 in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS).<br>2.Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) functional class from baseline to Week 30.<br>3.Change from baseline to Week 30 in N-terminal pro b-type natriuretic peptide (NT-proBNP).<br>4.Change from baseline to Week 30 in cardiac troponins.