Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy

NCT06947590 | Active Not Recruiting DMC

• 1 other identifier: MVLOHCM
• Study Type: interventional
• Target: 78
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

HCM - Hypertrophic Cardiomyopathy; Mavacamten

Keywords

HCM; Mavacamten

Outcome Measures

Primary Outcomes (1)

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

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

  From baseline to week 30.

Secondary Outcomes (7)

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

  At week 30.

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

  At week 30.

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

  At week 30.

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

  From baseline to week 30.

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

  From baseline to week 30.

Study Arms (2)

Mavacamten Group

EXPERIMENTAL

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.

Drug: mavacamten

Control Group

NO INTERVENTION

Patients in this group did not receive Mavacamten treatment and were instead managed with traditional therapies of their choice, such as beta-blockers or calcium channel blockers.

Interventions

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.

Mavacamten Group

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Xu Liu: lead

Study Sites (1)

Shanghai Chest Hospital

Shanghai, China

Location

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic

Interventions

MYK-461

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 20, 2025
• First Posted: April 27, 2025
• Study Start: April 20, 2025
• Primary Completion: February 1, 2026
• Study Completion: February 1, 2026
• Last Updated: April 27, 2025

Record last verified: 2025-04

Locations

CN (1)