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

NCT ID: NCT07103655

Last Updated: 2026-01-21

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 132 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-01
• Study Completion Date: 2027-01-01

Brief Summary

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.

Conditions

Hypertrophic Cardiomyopathy (HCM)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Mavacamten

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

Group Type: EXPERIMENTAL

Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol

Intervention Type: DRUG

Administer an appropriate dose of beta-blockers according to the patient's tolerance.

diltiazem

Intervention Type: DRUG

Administer an appropriate dose of diltiazem according to the patient's tolerance.

mavacamten

Intervention Type: DRUG

Add Mavacamten to guideline-directed standard medical therapy for patients with hypertrophic cardiomyopathy (HCM) and mid-to-apical left ventricular obstruction.

No mavacamten

Guideline-directed standard medical therapy group

Group Type: ACTIVE_COMPARATOR

Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol

Intervention Type: DRUG

Administer an appropriate dose of beta-blockers according to the patient's tolerance.

diltiazem

Intervention Type: DRUG

Administer an appropriate dose of diltiazem according to the patient's tolerance.

Interventions

Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol

Administer an appropriate dose of beta-blockers according to the patient's tolerance.

Intervention Type: DRUG

diltiazem

Administer an appropriate dose of diltiazem according to the patient's tolerance.

Intervention Type: DRUG

mavacamten

Add Mavacamten to guideline-directed standard medical therapy for patients with hypertrophic cardiomyopathy (HCM) and mid-to-apical left ventricular obstruction.

Intervention Type: DRUG

Other Intervention Names

beta receptor blockers

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Second Affiliated Hospital, School of Medicine, Zhejiang University

OTHER

Sponsor Role: lead

Responsible Party

xie xiaojie

MD, PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Second Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Countries

China

Central Contacts

Xiaojie Xie, MD, PhD

Role: CONTACT

xiexj@zju.edu.cn

571-87784700

Facility Contacts

Xiaojie Xie, MD, PhD

Role: primary

xiexj@zju.edu.cn

571-87784700

Other Identifiers

2025-1056

Identifier Type: -

Identifier Source: org_study_id