Serving the HCM Spectrum Disorder Community Since 1996

Clinical Trial - PIONEER-HCM

The HCMA is pleased to inform you of a clinical trial for those with HCM.

Trial name: A Phase 2 Open-label Pilot Study to Evaluate Efficacy, Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of MYK-461 in Subjects with Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

Ages Eligible for Study: 18 to 70 Years
Genders Eligible for Study: All
Accepts Healthy Volunteers: No


Key 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

Key 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 or history of paroxysmal atrial fibrillation with resting rate document > 100bpm within 1 year of 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 obstructive 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.
  • Part A: Ongoing therapy with beta blockers, calcium channel blockers, or disopyramide. Subjects on any of these medications who, in the opinion of the investigator, can safely be withdrawn are eligible as long as medication is discontinued at least 14 days prior to the Screening visit.
  • Part B: Ongoing therapy with calcium channel blockers or disopyramide. Subjects on any of these medications who, in the opinion of the investigator, can safely be withdrawn are eligible as long as medication is discontinued at least 14 days prior to the Screening visit.
  • 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.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies. 

Please refer to this study by its ClinicalTrials.gov identifier: NCT02842242
Contact: MyoKardia Clinical Team   clinicaltrials@myokardia.com  

United States, Arizona
Mayo Clinic Arizona Recruiting
Scottsdale, Arizona, United States
Principal Investigator: Steven Lester         
United States, Connecticut
Yale New Haven Hospital Recruiting
New Haven, Connecticut, United States
Principal Investigator: Daniel Jacoby         
United States, Massachusetts
Tufts Medical Center Recruiting
Boston, Massachusetts, United States
Principal Investigator: Martin Maron         
United States, Missouri
Washington University St. Louis Recruiting
Saint Louis, Missouri, United States
Principal Investigator: Richard Bach         
United States, North Carolina
Duke Health Center at Southpoint Recruiting
Durham, North Carolina, United States
Principal Investigator: Andrew Wang         
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States
Principal Investigator: Stephen Heitner         
United States, Pennsylvania
Hospital of the University of Pennsylvania (Penn Heart and Vascular Center) Recruiting
Philadelphia, Pennsylvania, United States
Principal Investigator: Kenneth Margulies         
Principal Investigator: Anjali Owens