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

Официальное название

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

Обзор

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

Вмешательства

• Препарат Mavacamten
  According to the product label

Первичные конечные точки

• Change in either resting or Valsalva Left Ventricular Outflow Tract (LVOT) peak gradient whichever used to judge the initiation of mavacamten treatment [Срок оценки: Baseline and up to week 16]

Вторичные конечные точки (12)

• Change in Left Ventricular Outflow Tract (LVOT) peak gradient (resting and Valsalva maneuver) [Срок оценки: 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) [Срок оценки: Baseline and up to week 16]
• Proportion of patients with any decrease in resting or Valsalva Left Ventricular Outflow Tract (LVOT) peak gradients [Срок оценки: Baseline and up to week 16]
• Proportion of patients with ≥1 New York Heart Association (NYHA) functional class improvement [Срок оценки: Baseline and up to week 16]
• Change in cardiac biomarkers from baseline [Срок оценки: 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) [Срок оценки: Baseline and up to week 16]
• Number of participants with Left Ventricular Ejection Fraction (LVEF) <50% as assessed by transthoracic echocardiography (TTE) [Срок оценки: Baseline and up to week 16]
• Change in left ventricular cardiac output (LVCO) from baseline assessed by transthoracic echocardiography (TTE) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: Baseline and up to week 16]
• Change in cardiac structural dimensions from baseline assessed by transthoracic echocardiography (TTE) [Срок оценки: 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) [Срок оценки: Baseline and up to week 16]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Япония · 1 центр

• Mebix. Inc — Minato-ku

Идентификаторы

NCT: NCT07541833 · CV027-1233

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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