A Study to Evaluate the Safety, Tolerability, Effectiveness, Biological Activity, and Drug/Body Interactions of MYK-461 in Patients with Symptomatic Enlargement of the Heart and Obstruction of Left Ventricle Outflow

Overview

About this study

The purpose of this study is to evaluate the safety, tolerability, effectiveness, biological activity, and drug/body interactions of MYK-461 in adult patients who have symptomatic enlargement of the heart and obstruction of left ventricle outflow.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria                   

  • Diagnosed with HCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15 mm at time of initial diagnosis or ≥ 13 mm with a positive family history of HCM
  • Age 18-70
  • BMI 18-37kg/m2
  • Documented LVEF ≥ 55% at the screening visit as determined by the investigator and the investigational site's echocardiography laboratory
  • Resting LVOT gradient ≥ 30 mg Hg and post-exercise peak LVOT gradient ≥ 50 mm Hg
  • NYHA functional class II or higher

Exclusion Criteria

  • History of sustained ventricular tachyarrhythmia
  • History of syncope with exercise within past 6 months
  • Active infection
  • Persistent atrial fibrillation or atrial fibrillation at screening
  • Has QTc Fridericia (QTcF) > 500 ms, or any other ECG abnormality considered by the investigator to pose a risk to subject safety (e.g. second degree atrioventricular block type II)
  • Aortic stenosis or fixed subaortic obstruction
  • History of LV systolic dysfunction (LVEF < 45%) at any time during their clinical course
  • History of coronary artery disease
  • History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator or MyoKardia physician, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion
  • Ongoing therapy with beta blockers, calcium channel blockers, or disopyramide
    • Subjects not on beta blockers, or on a stable dose of beta blockers and will require ongoing therapy can enroll in part B of study
    • Subjects in part A are required to washout from current treatment for atleast 14 days prior to screening
  • Prior treatment with cardiotoxic agents such as doxorubicin or similar, or current treatment with antiarrhythmic drugs that have negative inotropic activity, e.g. flecainide or propafenone
  • Prior participation in a clinical trial with MYK-461 not eligible for part B of study
  • Use of estrogen based hormonal contraception as highly effective contraceptive

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Steven Lester, M.D.

Closed for enrollment

More information

Publications

Publications are currently not available

Additional contact information

Non-cancer trials contact form

Phone: 800-664-4542 (toll-free)

International patient clinical studies questions