Cardiomyopathy
Conditions
Keywords
hypertrophic cardiomyopathy (HOCM)
Brief summary
The purpose of this study is to assess the real-world effectiveness and safety of mavacamten in adults diagnosed with symptomatic obstructive hypertrophic cardiomyopathy (HOCM) receiving cibenzoline in Japan
Interventions
DRUGMavacamten
According to the product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
• Signed informed consent form (ICF): Participants, or their legally acceptable representative, must have signed and dated an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved ICF in accordance with regulatory, local, and institutional guidelines. This must be obtained before the performance of any protocol-related procedures. * Diagnosed with obstructive hypertrophic cardiomyopathy (HOCM) consistent with Japanese Circulation Society guidelines (2025), i.e., satisfy all criteria below: * Has unexplained left ventricular (LV) hypertrophy with nondilated ventricular chambers in the absence of other cardiac (e.g., hypertension, aortic stenosis) or systemic disease and with maximal LV wall thickness ≥ 15 mm (or ≥ 13 mm with positive family history of HCM). * Has Left Ventricular Outflow Tract (LVOT) peak gradient ≥ 30 mmHg (resting, Valsalva maneuver, or post-exercise). * Has documented Left Ventricular Ejection Fraction (LVEF) ≥ 55% at baseline. * Participants who meet any of the following criteria: * Participants who have previously received mavacamten continuously for ≥ 16 weeks * Participants who are currently receiving mavacamten * Participants who are scheduled to receive mavacamten * Treated with a stable dose of cibenzoline for at least 3 months prior to initiating mavacamten treatment. Tapered cibenzoline within 3 months prior to initiating mavacamten treatment is allowed if stable dose of cibenzoline was used for at least 3 months prior to tapering. * At least 18 years of age at the time of signing the informed consent.
Exclusion criteria
* Hypersensitivity to the active substance or to any of the excipients. * During pregnancy and in women of childbearing potential. * Treated with strong CYP3A4 inhibitors (itraconazole, clarithromycin, voriconazole, posaconazole, ritonavir, cobicistat, ceritinib, ensitrelvir fumaric acid, lonafarnib, josamycin, or mifepristone/misoprostol). * Severe hepatic impairment (Child-Pugh C). * Severe atrioventricular block or severe sinoatrial block. * Congestive heart failure. * Requiring dialysis. * Angle-closure glaucoma. * Tendency to urinary retention. * Treated with vardenafil hydrochloride hydrate, moxifloxacin hydrochloride, lascufloxacin hydrochloride (injection), toremifene citrate, fingolimod hydrochloride, siponimod fumarate, or eliglustat tartrate. * Mavacamten treatment within 8 weeks prior to baseline. Mavacamten treatment initiation was judged based on post-exercise LVOT peak gradient.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in either resting or Valsalva Left Ventricular Outflow Tract (LVOT) peak gradient whichever used to judge the initiation of mavacamten treatment | Baseline and up to week 16 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Left Ventricular Outflow Tract (LVOT) peak gradient (resting and Valsalva maneuver) | Baseline and up to week 16 | — |
| Proportion of patients achieving target Left Ventricular Outflow Tract (LVOT) peak gradients (<50 mmHg / <30 mmHg) (Valsalva maneuver or post-exercise) | Baseline and up to week 16 | — |
| Proportion of patients with any decrease in resting or Valsalva Left Ventricular Outflow Tract (LVOT) peak gradients | Baseline and up to week 16 | — |
| Proportion of patients with ≥1 New York Heart Association (NYHA) functional class improvement | Baseline and up to week 16 | — |
| Change in cardiac biomarkers from baseline | Baseline and up to week 16 | Biomarkers include brain natriuretic peptide (BNP) or N-terminal prohormone of BNP (NT-proBNP) |
| Change in systolic function parameters (left ventricular ejection fraction (LVEF) and left ventricular fractional shortening (LVFS) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Number of participants with Left Ventricular Ejection Fraction (LVEF) <50% as assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in left ventricular cardiac output (LVCO) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in early diastolic mitral annular velocity (e') measured at the lateral, septal, and averaged positions from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in E/e' ratio measured at the lateral, septal, and averaged positions and E/A ratio (ratio of early (E) to late (A) ventricular filling velocities) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in cardiac structural dimensions from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | Structural dimensions include: septal thickness, posterior wall thickness, maximal left ventricular wall thickness, left ventricular end-diastolic diameter, left ventricular end-systolic diameter, and left atrial dimension |
| Change in left ventricular outflow tract (LVOT) gradient measured at rest and during Valsalva maneuver from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in cardiac chamber volumes (left ventricular end-diastolic volume, left ventricular end-systolic volume, and left atrial volume) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in indexed cardiac chamber volumes (left ventricular end-diastolic volume index, left ventricular end-systolic volume index, and left atrial volume index) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in left ventricular diameters (left ventricular end-diastolic diameter and left ventricular end-systolic diameter) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in indexed left ventricular diameters (left ventricular end-diastolic diameter index and left ventricular end-systolic diameter index) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in left atrial dimension from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in left ventricular mass index (LVMI) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Number of participants with systolic anterior motion (SAM) of the mitral valve, and number of participants with mitral regurgitation (MR) assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Number of treatment emergent adverse events (TEAEs)/adverse drug reactions (ADRs) and serious TEAEs/ADRs | Up to week 16 | — |
| Proportion of participants continuing cibenzoline | Baseline and weeks 4, 8, 12, and 16 | — |
| Proportion of patients who decrease or increase dose and/or frequency of cibenzoline, beta blocker, or calcium channel blocker | Baseline and weeks 4, 8, 12, and 16 | — |
| Daily dose of cibenzoline, beta blocker, or calcium channel blocker | Baseline and weeks 4, 8, 12, and 16 | — |
| Change in Left Ventricular Ejection Fraction (LVEF) as assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
| Change in left ventricular stroke volume (LVSV) from baseline assessed by transthoracic echocardiography (TTE) | Baseline and up to week 16 | — |
Countries
Japan
Contacts
CONTACTBMS Clinical Trials Contact Center www.BMSClinicalTrials.com
CONTACTFirst line of the email MUST contain NCT # and Site #.
STUDY_DIRECTORBristo Myers Squibb
Bristo Myers Squibb
Outcome results
None listed