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

NCT07103655 | Not Yet Recruiting Phase 4

• 1 other identifier: 2025-1056
• Study Type: interventional
• Target: 132
• Locations: 1 country
• Sites: 1

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)

Outcome Measures

Primary Outcomes (1)

• Percentage change in pressure gradient during the Valsalva maneuver

  The percentage reduction in the pressure gradient at the site of left ventricular obstruction during the Valsalva maneuver at week 36, compared to baseline, as measured by echocardiography.

  Week 36

Secondary Outcomes (11)

• Anterior papillary muscle to the interventricular septum distance

  Week 36

• Left atrial global longitudinal strain

  Week 36

• Left ventricular global longitudinal strain

  Week 36

• Percentage change in resting pressure gradient

  Week 36

• Absolute change in pressure gradient during the Valsalva maneuver

  Week 36

Study Arms (2)

Mavacamten

EXPERIMENTAL

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

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

No mavacamten

ACTIVE COMPARATOR

Guideline-directed standard medical therapy group

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

Interventions

diltiazem DRUG

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

Mavacamten; No mavacamten

mavacamten DRUG

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

Also known as: beta receptor blockers

Mavacamten

Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol DRUG

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

Mavacamten; No mavacamten

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Second Affiliated Hospital, School of Medicine, Zhejiang University: lead

Study Sites (1)

Second Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310009, China

Location

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic

Interventions

MYK-461; Adrenergic beta-Antagonists; Metoprolol; Bisoprolol; Carvedilol; Diltiazem

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases

Intervention Hierarchy (Ancestors)

Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Physiological Effects of Drugs; Phenoxypropanolamines; Propanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Propanols; Amines; Carbazoles; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 3-Ring; Benzazepines

Central Study Contacts

Xiaojie Xie, MD, PhD

CONTACT

571-87784700; xiexj@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: July 29, 2025
• First Posted: August 5, 2025
• Study Start: January 1, 2026
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: January 21, 2026

Record last verified: 2026-01

Locations

CN (1)