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

A Study Evaluating Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy Treated With Cibenzoline in Japan (MANAGE-HCM)

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 Overview

• Status: Recruiting
• Conditions: Cardiomyopathy
• Intervention / Treatment: Drug: Mavacamten

• Study Type: Observational
• Enrollment (Estimated): 36

Contacts and Locations

• Study Contact: Name: First line of the email MUST contain NCT # and Site #.
• Study Contact Backup: Name: BMS Clinical Trials Contact Center www.BMSClinicalTrials.com; Phone Number: 855-907-3286; Email: Clinical.Trials@bms.com

Study Locations

• Japan
  • Kochi, Japan
    • Recruiting
    • Kochi University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of adults diagnosed with symptomatic obstructive hypertrophic cardiomyopathy (HOCM) who have been prescribed mavacamten treatment by the treating physician as part of routine clinical care, in Japan

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Switch Group; Participants who discontinue cibenzoline before or at the initiation of mavacamten treatment	Drug: Mavacamten; According to the product label
Tapering/Add-on Group; Participants who continue cibenzoline at the initiation of mavacamten treatment	Drug: Mavacamten; According to the product label

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Biomarkers include brain natriuretic peptide (BNP) or N-terminal prohormone of BNP (NT-proBNP)	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)		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)	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	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)		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

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb
• Investigators: Study Director: Bristo Myers Squibb, Bristo Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

Study record dates

Study Major Dates

• Study Start (Actual): February 5, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: March 11, 2026
• First Submitted That Met QC Criteria: April 14, 2026
• First Posted (Actual): April 21, 2026

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: April 14, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: hypertrophic cardiomyopathy (HOCM)
• Additional Relevant MeSH Terms: Aortic Valve Disease; Cardiovascular Diseases; Heart Diseases; Heart Valve Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Cardiomyopathies; Cardiomyopathy, Hypertrophic; MYK-461
• Other Study ID Numbers: CV027-1233

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes