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

Zhejiang University

Status and phase: Not yet enrolling; Phase 4

Conditions: Hypertrophic Cardiomyopathy (HCM)

Treatments: Drug: mavacamten; Drug: diltiazem; Drug: Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol

Study type: Interventional

Funder types: Other

Identifiers

NCT07103655

2025-1056

Details and patient eligibility

About

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.

Enrollment: 132 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

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

  1. Left ventricular wall thickness ≥15 mm at end-diastole in any segment as assessed by echocardiography or cardiac magnetic resonance imaging (CMR);

  2. Left ventricular wall thickness ≥13 mm in individuals with a confirmed pathogenic gene mutation or in genetically affected family members;

  3. 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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  1. Presence of clinical symptoms such as dyspnea, chest pain, dizziness, palpitations, or syncope, with New York Heart Association (NYHA) functional class II-III;

  2. Maximal pressure gradient (PGmax) >30 mmHg in the mid-ventricle under resting or Valsalva maneuver as assessed by echocardiography;

  3. 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.

     Exclusion Criteria:

     -

• 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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132 participants in 2 patient groups

Mavacamten

Experimental group

Description:

Add-on use of mavacamten on top of guideline-directed standard medical therapy

Treatment:

Drug: Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol

Drug: diltiazem

Drug: mavacamten

No mavacamten

Active Comparator group

Description:

Guideline-directed standard medical therapy group

Treatment:

Drug: Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol

Drug: diltiazem