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

Not Applicable

Not yet recruiting

• Conditions: Hypertrophic Cardiomyopathy (HCM)
• Interventions: Drug: mavacamten; Drug: Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol; Drug: diltiazem

• Registration Number: NCT07103655
• Lead Sponsor: Second Affiliated Hospital, School of Medicine, Zhejiang University

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-07
• Study First Submitted: 2025-07-29
• Study First Submitted QC: 2025-07-29
• Last Update Submitted: 2025-07-29
• Study First Posted: 2025-08-05
• Last Update Posted: 2025-08-05
• Study Start: 2026-01-01
• Primary Completion: 2026-07-01
• Study Completion: 2027-01-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 132

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Mavacamten	mavacamten	Add-on use of mavacamten on top of guideline-directed standard medical therapy
Mavacamten	Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol	Add-on use of mavacamten on top of guideline-directed standard medical therapy
Mavacamten	diltiazem	Add-on use of mavacamten on top of guideline-directed standard medical therapy
No mavacamten	Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol	Guideline-directed standard medical therapy group
No mavacamten	diltiazem	Guideline-directed standard medical therapy group

Primary Outcome Measures

Name	Time	Method
Percentage change in pressure gradient during the Valsalva maneuver	Week 36	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.

Secondary Outcome Measures

Name	Time	Method
New York Heart Association (NYHA) functional classification	Week 36	The proportion of patients in the Mavacamten treatment group and the standard therapy group who achieved an improvement of ≥1 NYHA functional class from baseline at week 36.
Percentage change in resting pressure gradient	Week 36	The percentage reduction in the pressure gradient at the site of left ventricular obstruction at rest measured by echocardiography at week 36 compared to baseline.
Absolute change in pressure gradient during the Valsalva maneuver	Week 36	The absolute decrease in number of the pressure gradient at the site of left ventricular obstruction during the Valsalva maneuver measured by echocardiography at week 36 compared to baseline.
KCCQ-CSS	Week 36	The number increase in KCCQ-CSS questionnaire score at week 36 compared to baseline. The KCCQ-23 is a disease-specific health status assessment tool consisting of 23 items that quantify physical limitation, symptoms, self-efficacy, social limitation, and quality of life related to heart failure. Scores range from 0 to 100, with higher scores indicating better health status.
The proportion of patients with a pressure gradient <30 mmHg during the Valsalva maneuver	Week 36	The proportion of patients with a pressure gradient \<30 mmHg at the site of obstruction during the Valsalva maneuver at week 36.
BNP	Week 36	Number decrease in BNP levels from baseline to week 36.
Troponin T	Week 36	Number decrease in Troponin T levels from baseline to week 36.
New-onset atrial fibrillation	From baseline to week 36	Rate of new-onset atrial fibrillation in each group during the study period
LVEF<50%	From baseline to week 36	The proportion of patients in each group with LVEF \<50% accompanied by signs and symptoms of heart failure worsening and/or an increase in BNP of ≥30% compared to the previous measurement during the study period.
LVEF<40%	From baseline to week 36	Rate of LVEF \<40% in each group during the study period.

Trial Locations

Locations (1)

Second Affiliated Hospital, Zhejiang University School of Medicine; 🇨🇳 Hangzhou, Zhejiang, China