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A Phase 3, Open-label, Single Arm, Clinical Study to Evaluate Efficacy, Safety
and Tolerability of Mavacamten in Adults With Symptomatic Obstructive
Hypertrophic Cardiomyopathy

A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy
( HORIZON-HCM ) (CV027-004)

Florea Victoria

Bristol-Myers Squibb

1-2-1 Otemachi, Chiyoda-ku, Tokyo

mg-jp-clinical_trial@bms.com

Florea Victoria

Bristol-Myers Squibb

1-2-1 Otemachi, Chiyoda-ku, Tokyo

+81-120-093-507

MG-JP-RCO-JRCT@bms.com

Not Recruiting

June. 21, 2022

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

- 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

Both

Obstructive Hypertrophic Cardiomyopathy

Single Group Assignment: Mavacamten
Participants will receive treatment with 1, 2.5, 5, 10, 1mg dose once daily.

Change from Baseline to Week 30 in post exercise left ventricular outflow tract (LVOT) peak gradient as determined by Doppler echocardiography

1.Change from Baseline to Week 30 in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS).
2.Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) functional class from baseline to Week 30.
3.Change from baseline to Week 30 in N-terminal pro b-type natriuretic peptide (NT-proBNP).
4.Change from baseline to Week 30 in cardiac troponins.

+81-3-5550-7158

May. 19, 2022

No

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