Hypertrophic Cardiomyopathy Clinical Trial
A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
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.
Eligibility Criteria
Key Inclusion Criteria:
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 Exclusion Criteria:
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.
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There are 31 Locations for this study
Scottsdale Arizona, 85259, United States
Los Angeles California, 90048, United States
Palo Alto California, 94305, United States
San Francisco California, 94143, United States
New Haven Connecticut, 06520, United States
Evanston Illinois, 60208, United States
Indianapolis Indiana, 46260, United States
Iowa City Iowa, 52242, United States
Baltimore Maryland, 21201, United States
Boston Massachusetts, 02115, United States
Ann Arbor Michigan, 48109, United States
Detroit Michigan, 48202, United States
Saint Louis Missouri, 63110, United States
Mineola New York, 11501, United States
New York New York, 10016, United States
Charlotte North Carolina, 28203, United States
Durham North Carolina, 27713, United States
Cincinnati Ohio, 45219, United States
Portland Oregon, 97239, United States
Bethlehem Pennsylvania, 18018, United States
Hershey Pennsylvania, 17033, United States
Philadelphia Pennsylvania, 19104, United States
Pittsburgh Pennsylvania, 15213, United States
Dallas Texas, 75390, United States
Houston Texas, 77030, United States
Houston Texas, 77030, United States
Murray Utah, 84107, United States
Salt Lake City Utah, 84132, United States
Charlottesville Virginia, 22908, United States
Richmond Virginia, 23298, United States
Seattle Washington, 98195, United States
Madison Wisconsin, 53713, United States
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