Mavacamten use and effectiveness in adults with obstructive hypertrophic cardiomyopathy in Japan
A Study Evaluating Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy Treated With Cibenzoline in Japan (MANAGE-HCM)
This project will see how well mavacamten works and how safe it is for adults in Japan with symptomatic obstructive hypertrophic cardiomyopathy who are receiving cibenzoline.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 36 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Bristol-Myers Squibb Industry-sponsored |
| Drugs / interventions | ceritinib |
| Locations | 1 site (Kochi) |
| Trial ID | NCT07541833 on ClinicalTrials.gov |
What this trial studies
This is an observational, real-world study collecting treatment patterns, clinical outcomes, and safety data for adults with obstructive hypertrophic cardiomyopathy treated with mavacamten while on cibenzoline in Japan. Eligible patients meet the Japanese Circulation Society criteria for HOCM, including specified left ventricular wall thickness, LVOT gradient ≥ 30 mmHg, and LVEF ≥ 55%. Data will be gathered at participating sites such as Kochi University and include medication usage, symptom measures, imaging/gradient results, and adverse events. The sponsor is Bristol-Myers Squibb and the analysis will focus on effectiveness and safety in routine clinical practice rather than a randomized intervention.
Who should consider this trial
Good fit: Adults in Japan with symptomatic obstructive hypertrophic cardiomyopathy per Japanese Circulation Society criteria (LV wall thickness ≥15 mm or ≥13 mm with family history, LVOT peak gradient ≥30 mmHg, LVEF ≥55%) who are receiving cibenzoline and can provide informed consent.
Not a fit: Patients without obstructive HCM, with reduced LVEF, pediatric patients, or those not receiving cibenzoline are unlikely to match this study population or benefit from its specific findings.
Why it matters
Potential benefit: If successful, the results could help clinicians in Japan understand whether mavacamten is effective and safe for patients with obstructive HCM taking cibenzoline and guide treatment choices.
How similar studies have performed: Randomized trials such as EXPLORER-HCM and VALOR-HCM have shown clinical benefits of mavacamten in obstructive HCM, but real-world data in Japan, especially among patients on cibenzoline, remain limited.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Kochi
- Kochi University — Kochi, Japan (Recruiting)
Study contacts
- Study coordinator: BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
- Email: Clinical.Trials@bms.com
- Phone: 855-907-3286
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.