A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
- Conditions
- Non-obstructive Hypertrophic Cardiomyopathy
- Interventions
- Drug: mavacamtenDrug: Placebo
- Registration Number
- NCT03442764
- Lead Sponsor
- MyoKardia, Inc.
- Brief Summary
This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 59
- Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
- Age 18 and greater, Body weight > 45kg
- Documented LVEF ≥ 55% at the Screening as determined by echo central lab
- LVOT gradient < 30 mmHg at rest AND during Valsalva AND post-exercise
- NYHA functional class II or III
- Elevated NT-proBNP at rest
Key
- History of syncope, sustained ventricular tachyarrhythmia with exercise, obstructive coronary artery disease or myocardial infarction within the past 6 months
- History of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator (ICD) discharge within 6 months prior to Screening
- Current treatment with disopyramide or ranolazine (within 14 days prior to Screening)
- Current or planned treatment during the study with a combination of beta-blockers and calcium channel blockers
- Has been treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening
- History of resting or post-exercise LVOT >30 mmHg unless subsequently treated by septal reduction
- Has QTc Fridericia (QTcF) >480 ms or any other ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II)
- Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate-controlled within 1 year of Screening
- History of clinically significant malignant disease within 10 years such as non-metastatic cutaneous squamous cell or basal cell carcinoma
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group 1 mavacamten Active Treatment for participants with base target trough concentration Group 2 mavacamten Active Treatment for participants with higher target trough concentration Placebo Placebo Placebo Group
- Primary Outcome Measures
Name Time Method Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE) From first dose to 8 weeks following last dose (Up to 24 weeks) This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE) From first dose to 8 weeks following last dose (Up to 24 weeks) This is the percentage of participants who experienced at least one serious treatment-emergent adverse event (STEAE)
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (32)
The University of Texas Southwestern Medical Center at Dallas
🇺🇸Dallas, Texas, United States
UPMC Presbyterian
🇺🇸Pittsburgh, Pennsylvania, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
Mayo Clinic Arizona
🇺🇸Scottsdale, Arizona, United States
Northwestern University
🇺🇸Evanston, Illinois, United States
Cedars-Sinai Medical Center (Smidt Heart Institute)
🇺🇸Los Angeles, California, United States
St. Vincent Medical Group
🇺🇸Indianapolis, Indiana, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
University of Cincinnati Medical Center
🇺🇸Cincinnati, Ohio, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Intermountain Medical Center
🇺🇸Murray, Utah, United States
University of Virginia
🇺🇸Charlottesville, Virginia, United States
Unity Point Health Meriter Heart and Vascular Institute
🇺🇸Madison, Wisconsin, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
Stanford Hospital and Clinics/Stanford University
🇺🇸Palo Alto, California, United States
University of Iowa Hospitals and clinics
🇺🇸Iowa City, Iowa, United States
University of Maryland Medical System
🇺🇸Baltimore, Maryland, United States
St. Luke's Cardiology Associates
🇺🇸Bethlehem, Pennsylvania, United States
NYU Langone Medical Center
🇺🇸New York, New York, United States
Carolinas Medical Center
🇺🇸Charlotte, North Carolina, United States
Houston Methodist Hospital
🇺🇸Houston, Texas, United States
Penn State Health Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
NYU Winthrop Hospital
🇺🇸Mineola, New York, United States
Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
🇺🇸Houston, Texas, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States
Duke Cardiology at Southpoint
🇺🇸Durham, North Carolina, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
University of Utah Medical Center
🇺🇸Salt Lake City, Utah, United States