Phase 3
N=38
A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy
Cardiomyopathy, Hypertrophic Obstructive
Bottom Line
View on ClinicalTrials.gov: NCT05414175 ↗Enrolled (actual)
38
Serious AEs
15.8%
Results posted
Dec 2024
Primary outcome: Primary: Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30 — -60.6963 millimeters of mercury (mmHg)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Mavacamten (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Bristol-Myers Squibb
- Primary completion
- Nov 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30 |
-60.6963 | — |
| SECONDARY Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30 |
9.766 | — |
| SECONDARY Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30 |
63.2 | — |
| SECONDARY Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30 |
-738.0 | — |
| SECONDARY Change From Baseline in Cardiac Troponin I at Week 30 |
-10.920 | — |
| SECONDARY Change From Baseline in Cardiac Troponin T at Week 30 |
-4.95 | — |
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).
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT05414175). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.