Symptomatic Obstructive Hypertrophic Cardiomyopathy
Conditions
Brief summary
The purpose of this study is to evaluate the safety, tolerability and effectiveness of mavacamten in adults with obstructive hypertrophic cardiomyopathy in India.
Interventions
DRUGMavacamten
Specified dose on specified days
Sponsors
Bristol-Myers Squibb
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosed with obstructive hypertrophic cardiomyopathy (HCM) consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines. * Has unexplained LV hypertrophy with nondilated ventricular chambers in the absence of other cardiac (ie, hypertension, aortic stenosis) or systemic disease and with maximal LV wall thickness ≥ 15 mm (or ≥ 13 mm with positive family history of hypertrophic cardiomyopathy). * Has LVOT (Valsalva left ventricular outflow tract) peak gradient ≥ 50 mmHg during screening as assessed by TTE at rest or with Valsalva maneuver. * Has LVOT peak gradient with Valsalva maneuver at screening TTE of ≥ 30 mmHg. * Has adequate acoustic windows to enable accurate TTEs. * Has New York Heart Association (NYHA) Class II or III symptoms at screening. * Body weight is greater than 45 kg at screening. * Documentation of LVEF ≥ 55% at rest of screening TTE.
Exclusion criteria
* Known infiltrative or storage disorder causing cardiac hypertrophy that mimics obstructive hypertrophic cardiomyopathy, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy. * Has paroxysmal atrial fibrillation present per the investigator's evaluation of the participant's ECG at the time of screening. * Has 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. (Note: Participants with persistent or permanent atrial fibrillation who are anticoagulated and adequately rate-controlled are allowed). * Has a history of syncope with exercise within 6 months prior to screening. * History of sustained ventricular tachyarrhythmia (\> 30 seconds) within 6 months prior to screening. * Has documented obstructive coronary artery disease (\> 70% stenosis in one or more epicardial coronary arteries) or history of myocardial infarction. * Other protocol-defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of serious Treatment Emergent Adverse Events (TEAEs) | Up to Week 48 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with major adverse cardiac events | Up to Week 48 | Major adverse cardiac events includes cardiovascular \[CV\] death, non-fatal stroke, nonfatal myocardial infarction and hospitalization for heart failure |
| Number of participants with CV hospitalization | Up to Week 48 | — |
| Number of participants with heart failure (HF) events | Up to Week 48 | HF includes HF related hospitalizations and urgent emergency room \[ER\]/outpatient visits |
| Number of participants with atrial fibrillation/flutter | Up to Weeks 48 | — |
| Number of participants with syncope | Up to Week 48 | — |
| Number of participants with left ventricular ejection fraction (LVEF) < 50% | Up to Week 30 | — |
| Number of participants with left ventricular ejection fraction (LVEF) < 45% | Up to Week 30 | — |
| Number of participants with left ventricular ejection fraction (LVEF) < 40% | Up to Week 30 | — |
| Number of participants with left ventricular ejection fraction (LVEF) < 30% | Up to Week 30 | — |
| Number of participants with non-serious AEs | Up to Week 48 | — |
| Change from baseline to Week 30 in Valsalva left ventricular outflow tract (LVOT) peak gradient | Up to Week 30 | — |
| Change from baseline to Week 30 in resting LVOT peak gradient | Up to Week 30 | — |
| Change from baseline to Week 30 in hs-troponin-I | Up to Week 30 | — |
| Change from baseline to Week 30 in N-terminal pro b-type natriuretic peptide (NT-proBNP) | Up to Week 30 | — |
| 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. |
Countries
India
Contacts
STUDY_DIRECTORBristol-Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed