The Therapeutic Value of Mavacamten in Hypertrophic Cardiomyopathy With Mid-to-Apical Left Ventricular Obstruction

Официальное название

The Therapeutic Value of Mavacamten in Hypertrophic Cardiomyopathy With Mid-to-Apical Left Ventricular Obstruction: A Prospective, Interventional, Real-World Clinical Study.

Обзор

This study is a prospective interventional cohort study aimed at evaluating the therapeutic efficacy and clinical utility of Mavacamten-a targeted myosin inhibitor specifically developed for obstructive hypertrophic cardiomyopathy (HCM)-in patients with HCM characterized by mid-to-apical left ventricular obstruction.

Вмешательства

• Препарат mavacamten
  Add Mavacamten to guideline-directed standard medical therapy for patients with hypertrophic cardiomyopathy (HCM) and mid-to-apical left ventricular obstruction.
• Препарат Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol
  Administer an appropriate dose of beta-blockers according to the patient's tolerance.
• Препарат diltiazem
  Administer an appropriate dose of diltiazem according to the patient's tolerance.

Первичные конечные точки

• Percentage change in pressure gradient during the Valsalva maneuver [Срок оценки: Week 36]

Вторичные конечные точки (11)

• Anterior papillary muscle to the interventricular septum distance [Срок оценки: Week 36]
• Left atrial global longitudinal strain [Срок оценки: Week 36]
• Left ventricular global longitudinal strain [Срок оценки: Week 36]
• Percentage change in resting pressure gradient [Срок оценки: Week 36]
• Absolute change in pressure gradient during the Valsalva maneuver [Срок оценки: Week 36]
• The proportion of patients with a pressure gradient <30 mmHg during the Valsalva maneuver [Срок оценки: Week 36]
• BNP [Срок оценки: Week 36]
• Troponin T [Срок оценки: Week 36]
• New-onset atrial fibrillation [Срок оценки: From baseline to week 36]
• LVEF<50% [Срок оценки: From baseline to week 36]
• LVEF<40% [Срок оценки: From baseline to week 36]

Критерии участия

Критерии включения

• Patients diagnosed with HCM according to the 2023 Chinese Guidelines for the Diagnosis and Treatment of Adult Hypertrophic Cardiomyopathy, meeting one of the following:

• Left ventricular wall thickness ≥15 mm at end-diastole in any segment as assessed by echocardiography or cardiac magnetic resonance imaging (CMR);
• Left ventricular wall thickness ≥13 mm in individuals with a confirmed pathogenic gene mutation or in genetically affected family members;
• Exclusion of other cardiovascular, systemic, or metabolic disorders that may cause ventricular hypertrophy.

• Symptomatic non-outflow tract obstructive HCM patients (meeting criterion a and at least one of b or c):

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• Presence of clinical symptoms such as dyspnea, chest pain, dizziness, palpitations, or syncope, with New York Heart Association (NYHA) functional class II-III;
• Maximal pressure gradient (PGmax) >30 mmHg in the mid-ventricle under resting or Valsalva maneuver as assessed by echocardiography;
• PGmax >30 mmHg in the apical region under resting or Valsalva maneuver on echocardiography.

③Ability to provide written informed consent (ICF) and any required privacy authorization prior to study enrollment.

Критерии исключения

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• Obstructive hypertrophic cardiomyopathy (HCM), defined as a maximal left ventricular outflow tract pressure gradient (LVOT-PGmax) ≥30 mmHg at rest and during the Valsalva maneuver on echocardiography;

• Maximal right ventricular outflow tract pressure gradient (RVOT-PGmax) ≥16 mmHg at rest; ③ Left ventricular ejection fraction (LVEF) <50% on echocardiography;

• Uncontrolled primary hypertension;

• Moderate or severe aortic valve stenosis and/or primary mitral valve disease with severe mitral regurgitation; ⑥ Known infiltrative or storage disorders mimicking the HCM phenotype (e.g., Fabry disease, cardiac amyloidosis); ⑦ Presence of severe infections, hepatic dysfunction, renal impairment, or other serious conditions significantly affecting life expectancy.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Second Affiliated Hospital, Zhejiang University School of Medicine — Ханчжоу

Идентификаторы

NCT: NCT07103655 · 2025-1056

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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