Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06947590

Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy

This study aimed to evaluate the efficacy and safety of Mavacamten compared to no treatment in patients with symptomatic latent obstructive hypertrophic cardiomyopathy. The trial was randomized into two groups: Mavacamten group and Non-Mavacamten group. Over the 30-week treatment period, patients underwent a series of assessments at predefined time points, including transthoracic echocardiography, electrocardiogram (ECG), Holter monitoring, NYHA functional classification, Kansas City Cardiomyopathy Questionnaire (KCCQ), and cardiac biomarkers.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

HCM - Hypertrophic Cardiomyopathy Aortic Stenosis, Subvalvular Aortic Valve Disease Aortic Valve Stenosis Cardiomyopathies Cardiomyopathy, Hypertrophic Cardiovascular Diseases Heart Diseases Heart Valve Diseases Mavacamten

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Shanghai Chest Hospital

Shanghai, China

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Age ≥18 years.
  • Weight greater than 45 kg.
  • Adequate acoustic windows to allow for accurate transthoracic echocardiograms (TTEs).
  • Diagnosis of latent obstructive hypertrophic cardiomyopathy, in accordance with the current guidelines of the American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Chinese Society of Cardiology.
  • Left ventricular ejection fraction (LVEF) ≥55% at rest, confirmed by the echocardiography core laboratory.
  • New York Heart Association (NYHA) Class II or III symptoms at the time of screening.
  • Resting oxygen saturation ≥90% at the time of screening.

Exclusion criteria

  • Any acute or severe comorbidities (e.g., severe infections or hematological, renal, metabolic, gastrointestinal, or endocrine dysfunction).
  • Currently using or having used prohibited medications within 14 days prior to screening, such as cytochrome CYP2C19 inhibitors (e.g., omeprazole or esomeprazole) or strong CYP3A4 inhibitors.
  • Life expectancy of less than 1 year.
  • Pregnant or breastfeeding women.
  • History of syncope or sustained ventricular tachyarrhythmia during exercise within the past 6 months.
  • Atrial fibrillation (AF).
  • Patients currently receiving or planning to receive treatment with disopyramide, cibenzoline, ranolazine, or a combination of beta-blockers with verapamil or diltiazem.

Treatment and study plan

Mavacamten

Drug

Patients in this group were treated with Mavacamten, starting at an initial dose of 2.5 mg, administered orally once daily. Subsequent doses were adjusted based on changes in pressure gradients and cardiac function observed during follow-up.

Primary outcomes

  1. The change in peak left ventricular outflow tract (LVOT) gradient

    Time frame: From baseline to week 30.

    The change in peak left ventricular outflow tract (LVOT) gradient, determined by Doppler echocardiography, during exercise or pharmacologic provocation from baseline to week 30.

Secondary outcomes

  1. The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 30 mmHg.

    Time frame: At week 30.

    The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 30 mmHg during exercise or pharmacologic provocation at week 30.

  2. The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg.

    Time frame: At week 30.

    The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg during exercise or pharmacologic provocation at week 30.

  3. The proportion of patients with at least a one-class improvement in NYHA functional classification.

    Time frame: At week 30.

    The proportion of patients with at least a one-class improvement in NYHA functional classification at week 30.

  4. The change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).

    Time frame: From baseline to week 30.

    The change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) from baseline to week 30.

  5. The change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels.

    Time frame: From baseline to week 30.

    The change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels from baseline to week 30.

  6. The change in high-sensitivity cardiac troponin I (hs-cTnI) levels.

    Time frame: From baseline to week 30.

    The change in high-sensitivity cardiac troponin I (hs-cTnI) levels from baseline to week 30.

  7. Key safety endpoints

    Time frame: From baseline to week 30.

    Key safety endpoints include the incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).

Sponsors and collaborators

Lead sponsor

Xu Liu

Other

Registry information

Official study title

Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy: A Randomized Controlled Trial

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Apr 27, 2025
Registry last updated
Apr 27, 2025

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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.