Mavacamten use and effectiveness in adults with obstructive hypertrophic cardiomyopathy in Japan

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Mebix. Inc
  Minato-ku, Tokyo, Japan Recruiting

Study contacts

• Study coordinator: BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
• Email: Clinical.Trials@bms.com
• Phone: 855-907-3286

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.