NCT05414175CompletedPhase 3
A Phase 3, Open-label, Single Arm, Clinical Study to Evaluate Efficacy, Safety and Tolerability of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Bristol-Myers Squibb20 sites in 1 country38 target enrollmentStarted: August 19, 2022Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 38
- Locations
- 20
- Primary Endpoint
- Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
Study Overview
Brief Summary
The purpose of this study is to evaluate the effectiveness, safety, and tolerability of a 30-week course of mavacamten and the long-term effects of mavacamten in Japanese participants with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •Age 18 and greater, body weight ≥ 35kg
- •Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
- •Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation Society guidelines
- •Has documented left ventricular ejection fraction (LVEF) ≥60% NYHA Class II or III
Exclusion Criteria
- •Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
- •History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
- •History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
- •Paroxysmal atrial fibrillation with atrial fibrillation present at the time of Screening.
- •Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
- •Treatment (within 14 days prior to Screening) or planned treatment during the study with cibenzoline, disopyramide or ranolazine
- •Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
- •Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study
- •ICD placement within 2 months prior to Screening or planned ICD placement during the study
- •Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation procedures, or completion
- •Prior treatment with cardiotoxic agents such as doxorubicin or similar
- •Other protocol-defined inclusion/exclusion criteria apply
Arms & Interventions
Mavacamten
Experimental
Intervention: Mavacamten (Drug)
Outcomes
Primary Outcomes
Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
Time Frame: At Baseline and Week 30
The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the doppler echocardiography. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
Secondary Outcomes
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30(At Baseline and Week 30)
- Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30(Baseline and at Week 30)
- Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30(At baseline and week 30)
- Change From Baseline in Cardiac Troponin I at Week 30(At baseline and week 30)
- Change From Baseline in Cardiac Troponin T at Week 30(At baseline and week 30)
Investigators
Study Sites (20)
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