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NCT Number: NCT07807384

Efficacy and Safety of an LVEF-Guided Mavacamten Dose Up-Titration Strategy in Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

This study aims to evaluate whether, under a treatment strategy primarily guided by left ventricular ejection fraction (LVEF), dose up-titration in adult patients with symptomatic obstructive hypertrophic cardiomyopathy who have received Mavacamten treatment for at least 12 weeks with a stable maintenance dose (as baseline) and with LVEF ≥55% can further reduce left ventricular outflow tract (LVOT) gradient after 24 weeks of treatment. It will also assess changes in hemodynamics, clinical symptoms, cardiac structure, and function. Furthermore, the study will conduct stratified analyses based on patients' baseline resting or Valsalva-provoked LVOT gradient levels at enrollment [<30 mmHg vs. ≥30 mmHg] to explore differences in the aforementioned outcome measures among patients with different gradient levels, with the goal of providing evidence for individualized clinical dose adjustment.

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Key information

Conditions

Obstructive Hypertrophic Cardiomyopathy (oHCM) Aortic Stenosis, Subvalvular Aortic Valve Disease Aortic Valve Stenosis Cardiomyopathies Cardiomyopathy, Hypertrophic Cardiovascular Diseases Heart Diseases Heart Valve Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 1. Adult patients diagnosed with obstructive hypertrophic cardiomyopathy (HCM) and classified as New York Heart Association (NYHA) functional class II-III. 2. Have received stable Mavacamten treatment for at least 12 weeks with a stable dose. 3. LVEF ≥ 55%.

Exclusion criteria

  • 1. Hypertrophy caused by other systemic diseases with a clear etiology. 2. Planned use of strong CYP2C19 inhibitors, moderate to strong CYP2C19 inducers, or CYP3A4 inducers during the study. 3. Planned use of disopyramide, ranolazine, verapamil combined with beta-blockers, or diltiazem combined with beta-blockers during the study. 4. Previous intolerance to Mavacamten. 5. Severe hepatic or renal dysfunction. 6. Pregnant or lactating women.

Treatment and study plan

Mavacamten

Drug

Eligible participants will undergo dose escalation at baseline from their current dose to the next higher level (once daily: 2.5 mg → 5 mg, 5 mg → 10 mg, or 10 mg → 15 mg).

At Week 4, clinical status and echocardiography will be re-evaluated, and the same dose will be maintained for the subsequent 8 weeks, unless LVEF is < 50%.

After 12 weeks of treatment at the same dose level:

If LVEF remains ≥ 55%, further dose escalation is permitted. If LVEF is between 50% and 55%, the current dose will be maintained. If LVEF is < 50%, treatment will be interrupted/discontinued.

Primary outcomes

  1. Change From Baseline in Valsalva Peak Left Ventricular Outflow Tract (LVOT) Pressure Gradient Assessed by Echocardiography at Week 24

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

Secondary outcomes

  1. Left Ventricular Wall Thickness

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  2. Left Atrial Volume Index

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  3. Left Ventricular Mass Index

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  4. Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)

    Time frame: Baseline and Week 24

    The Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) includes the physical limitation and total symptom domains. Scores range from 0 to 100, where higher scores indicate better health status, fewer symptoms, and less physical limitation (a better outcome).

  5. Mitral Regurgitation Severity

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  6. Systolic Anterior Motion

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  7. E/A Ratio

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  8. Left Ventricular Ejection Fraction

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  9. Left Atrial Volume Index

    Time frame: Baseline and Week 24

  10. Left Ventricular Wall Thickness

    Time frame: Baseline and Week 24

  11. New York Heart Association (NYHA) Functional Class

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

    Assessed by the New York Heart Association (NYHA) Functional Classification. The classification categorizes heart failure severity into four classes: Class I (no limitation of physical activity), Class II (slight limitation), Class III (marked limitation), and Class IV (unable to carry on any physical activity without discomfort). Scores range from Class I to Class IV, where higher classes indicate worse heart failure symptoms (a worse outcome)

  12. NT-proBNP

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

  13. Myocardial troponin

    Time frame: Baseline, Week 4, Week 8, Week 12, and Week 24

Study contacts

Contact information is provided by the study sponsor or research team.

Ping Li

CONTACT

[email protected]

0791-86300545

Sponsors and collaborators

Lead sponsor

Second Affiliated Hospital of Nanchang University

Other

Registry information

Official study title

Efficacy and Safety of a Left Ventricular Ejection Fraction-Guided Mavacamten Dose Up-Titration Strategy in Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy: A Prospective, Multicenter, Open-Label Study

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
Sep 8, 2026
Registry last updated
Sep 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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