HORIZON-HCM: A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy
Study Details
Study Description
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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Mavacamten
|
Drug: Mavacamten
Specified dose on specified days
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Change from Baseline to Week 30 in post exercise left ventricular outflow tract (LVOT) peak gradient as determined by Doppler echocardiography [Up to Week 30]
Secondary Outcome Measures
- Change from Baseline to Week 30 in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS) [Up to Week 30]
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a patient reported outcome instrument with minimum score = 0 and maximum score = 100 where higher score indicates better health status. The instrument utilizes a recall period of 2 weeks over which patients describe the frequency and severity of their symptoms, their physical and social limitations, and how they perceive their heart failure symptoms to affect their quality of life. The KCCQ clinical summary score (KCCQ-CSS) combines the physical limitation and total symptom scores.
- Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) functional class from baseline to Week 30 [Up to Week 30]
The New York Heart Association (NYHA) functional classification of heart failure assigns participants to 1 of 4 categories based on the participant's symptoms. Heart failure classification will be assessed by the Investigator at specified timepoints in the study. NYHA class at Week 30 will be compared to baseline and the proportion of participants with an improvement of at least one class will be determined.
- Change from baseline to Week 30 in N-terminal pro b-type natriuretic peptide (NT-proBNP) [Up to Week 30]
- Change from baseline to Week 30 in cardiac troponins [Up to Week 30]
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Local Institution - 0009 | Chuo-Ku | Tokyo | Japan | 104-0044 |
Sponsors and Collaborators
- Bristol-Myers Squibb
Investigators
- Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb
Study Documents (Full-Text)
None provided.More Information
Additional Information:
- BMS Clinical Trial Information
- BMS Clinical Trial Patient Recruiting
- FDA Safety Alerts and Recalls
- Investigator Inquiry Form
Publications
None provided.- CV027-004