Health Condition 1: I422- Other hypertrophic cardiomyopathy
Conditions
Interventions
Intervention1: Mavacamten capsules: Frequency - Once daily. Route - oral. Maximum duration of treatment - 120 weeks. All participants will start the double-blind treatment with mavacamten 5 mg or plac
Sponsors
MyoKardia, Inc.
Eligibility
Inclusion criteria
Inclusion criteria: 1) Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines: unexplained left-ventricular hypertrophy with non-dilated ventricular chambers in the absence of other cardiac or systemic disease which can produce the required magnitude of hypertrophy of a maximal LV wall thickness greater than or equal to 15 mm. 2) Peak LVOT pressure gradient 90% at screening. 7) NT-proBNP greater than or equal to 200 pg/mL or BNP greater than or equal to 70 pg/mL. 8) Evidence of myocardial damage or Evidence of LV diastolic dysfunction.
Exclusion criteria
Exclusion criteria: 1) Known infiltrative or storage disorder causing cardiac hypertrophy that mimics nHCM. 2) History of unexplained syncope within 6 months prior to screening. 3) History of sustained ventricular tachyarrhythmia ( > 30 seconds) within 6 months prior to screening. 4) Paroxysmal or persistent (non-permanent) AF detected at the time of screening. 5) ICD placement or pulse generator change within 2 months prior to screening or planned new ICD placement during the study. 6) Acute heart failure from 4 weeks prior to screening up to randomization. 7) Coronary artery disease requiring intervention. 8) Heart transplant recipient or listed for heart transplant. 9) Currently implanted LV assist device. 10) Clinically significant pulmonary disease associated with exertional dyspnea. 11) Any documented active or suspected malignancy or history of malignancy within 2 years prior to screening. 12) Clinically documented LV aneurysm greater than or equal to 2 cm
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1) To assess the efficacy of a 48-week course of mavacamten compared to placebo on patient reported health status 2) To assess the efficacy of a 48-week course of mavacamten compared to placebo on exercise capacity Timepoint: 1) Change from baseline in KCCQ-23 CSS at Week 4 2) Change from baseline in pVO2 at Week 48 | — |
Secondary
| Measure | Time frame |
|---|---|
| Evaluate the effects of mavacamten on ventilatory efficiency as measured by the VE/VCO2 slopeTimepoint: Change from baseline in VE/VCO2 slope to Week 48;Evaluate the effects of mavacamten on cardiac biomarkers of myocardial injuryTimepoint: Change from baseline in cTn-T to Week 48;Evaluate the effects of mavacamten on cardiac biomarkers of wall stressTimepoint: Change from baseline in NT-proBNP to Week 48;Evaluate the effects of mavacamten on composite of cardiovascular eventTimepoint: Time to first MACE-plus events defined as any CV death, non-fatal myocardial infarction, non-fatal stroke, hospitalization for heart failure, hospitalization for arrhythmias, or appropriate ICD therapy;Evaluate the effects of mavacamten on NYHA classificationTimepoint: Proportion of participants with at least 1 class of NYHA improvement from baseline to Week 48;Evaluate the effects of mavacamten on patient reported shortness of breathTimepoint: Change from baseline in HCMSQ SoB domain to Week 48 | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, Czech Republic, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Netherlands, Norway, Poland, Portugal, Spain, United Kingdom, United States of America
Contacts
Public ContactShilpi Sinha
Bristol Myers Squibb
Outcome results
None listed