Status:

COMPLETED

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

Lead Sponsor:

MyoKardia, Inc.

Conditions:

Obstructive Hypertrophic Cardiomyopathy

Eligibility:

All Genders

18+ years

Phase:

PHASE3

Brief 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 ...

Eligibility Criteria

Inclusion

  • Key
  • 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

  • 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

Key Trial Info

Start Date :

May 29 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

May 6 2020

Estimated Enrollment :

251 Patients enrolled

Trial Details

Trial ID

NCT03470545

Start Date

May 29 2018

End Date

May 6 2020

Last Update

October 4 2021

Active Locations (71)

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Page 1 of 18 (71 locations)

1

Mayo Clinic Arizona

Scottsdale, Arizona, United States, 85259

2

Cedars-Sinai Medical Center (Smidt Heart Institute)

Los Angeles, California, United States, 90048

3

UCSF School of Medicine

San Francisco, California, United States, 94143

4

Stanford University

Stanford, California, United States, 94305

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