Physician

Dr. Jamshid Shirani

Study Coordinator

Susan Hahn

484-526-7825

susan.hahn2@sluhn.org

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Synopsis: The purpose of this study is to compare the effect of the investigational drug, mavacamten, to a placebo (an inactive drug). This is being done to confirm that in obstructive Hypertrophic Cardiomyopathy (HCM) patients mavacamten is safe, and helps to improve symptoms or ability to exercise.

Mavacamten is an investigational drug, referred to as “study drug” in this consent form. This means that it has not been approved by the Food and Drug Administration (FDA) or any regulatory agency for routine clinical use, including the treatment of HCM.

MyoKardia Inc. is the sponsor of this study, also referred to as “MyoKardia.” MyoKardia is providing the study drug and is compensating the study doctor and site staff to conduct the study.

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• Age 18 and greater, body weight ≥ 45kg
• Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
• Dagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines and satisfy both criteria:H
• Has documented left ventricular ejection fraction (LVEF) ≥55%
• NYHA Class II or III
• Has documented oxygen saturation at rest ≥90% at Screening
• Is able to perform an upright CPET and has a respiratory exchange ratio (RER) ≥1.0 at Screening per central reading

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• 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 life-threatening ventricular arrhythmia within 6 months prior to Screening
• Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at 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 disopyramide or ranolazine
• Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of β-blockers and calcium channel blockers
• LVOT gradient with Valsalva maneuver <30 mmHg at Screening
• 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 6 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

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