Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan (MANAGE-HCM)

Bristol-Myers Squibb36 enrolled

Overview

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

Study Type

OBSERVATIONAL

Enrollment

Conditions

Cardiomyopathy

Interventions

MavacamtenDRUG

According to the product label

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Kochi University

Kochi, Japan

RECRUITING

Outcomes

Primary Outcomes

Change in either resting or Valsalva Left Ventricular Outflow Tract (LVOT) peak gradient whichever used to judge the initiation of mavacamten treatment

Time frame: Baseline and up to week 16

Secondary Outcomes

Change in Left Ventricular Outflow Tract (LVOT) peak gradient (resting and Valsalva maneuver)

Time frame: 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)

Time frame: Baseline and up to week 16

Proportion of patients with any decrease in resting or Valsalva Left Ventricular Outflow Tract (LVOT) peak gradients

Time frame: Baseline and up to week 16

Proportion of patients with ≥1 New York Heart Association (NYHA) functional class improvement

Time frame: Baseline and up to week 16

Change in cardiac biomarkers from baseline

Biomarkers include brain natriuretic peptide (BNP) or N-terminal prohormone of BNP (NT-proBNP)

Time frame: Baseline and up to week 16

Change in systolic function parameters (left ventricular ejection fraction (LVEF) and left ventricular fractional shortening (LVFS) from baseline assessed by transthoracic echocardiography (TTE)

Time frame: Baseline and up to week 16

Number of participants with Left Ventricular Ejection Fraction (LVEF) <50% as assessed by transthoracic echocardiography (TTE)

Time frame: Baseline and up to week 16

Central Contacts

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CONTACT

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CONTACT

Data from ClinicalTrials.gov

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Change in left ventricular cardiac output (LVCO) from baseline assessed by transthoracic echocardiography (TTE)

Time frame: 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)

Time frame: 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)

Time frame: Baseline and up to week 16

Change in cardiac structural dimensions from baseline assessed by transthoracic echocardiography (TTE)

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

Time frame: Baseline and up to week 16

Change in left ventricular outflow tract (LVOT) gradient measured at rest and during Valsalva maneuver from baseline assessed by transthoracic echocardiography (TTE)

Time frame: 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)

Time frame: 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)

Time frame: 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)

Time frame: 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)

Time frame: Baseline and up to week 16

Change in left atrial dimension from baseline assessed by transthoracic echocardiography (TTE)

Time frame: Baseline and up to week 16

Change in left ventricular mass index (LVMI) from baseline assessed by transthoracic echocardiography (TTE)

Time frame: 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)

Time frame: Baseline and up to week 16

Number of treatment emergent adverse events (TEAEs)/adverse drug reactions (ADRs) and serious TEAEs/ADRs

Time frame: Up to week 16

Proportion of participants continuing cibenzoline

Time frame: 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

Time frame: Baseline and weeks 4, 8, 12, and 16

Daily dose of cibenzoline, beta blocker, or calcium channel blocker

Time frame: Baseline and weeks 4, 8, 12, and 16

Change in Left Ventricular Ejection Fraction (LVEF) as assessed by transthoracic echocardiography (TTE)

Time frame: Baseline and up to week 16

Change in left ventricular stroke volume (LVSV) from baseline assessed by transthoracic echocardiography (TTE)

Time frame: Baseline and up to week 16