Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy

• Status: Completed
• Trial end date: 2020-05-06
• Target enrollment: 0
• Participant gender: All

Summary

This is a multicenter, international, double-blind study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM). Approximately 220 participants will be randomized to receive placebo or mavacamten.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: MyoKardia, Inc.

Criteria

Key Inclusion Criteria:

• Age 18 and greater, body weight ≥ 45kg

• Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)

• Diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines and satisfy both criteria:

• 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

Key Exclusion Criteria:

• 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 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