Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy

N/AActive Not RecruitingNCT06947590

Xu Liu78 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

HCM - Hypertrophic Cardiomyopathy Mavacamten

Interventions

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.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Age ≥18 years. 2. Weight greater than 45 kg. 3. Adequate acoustic windows to allow for accurate transthoracic echocardiograms (TTEs). 4. 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. 5. Left ventricular ejection fraction (LVEF) ≥55% at rest, confirmed by the echocardiography core laboratory. 6. New York Heart Association (NYHA) Class II or III symptoms at the time of screening. 7. Resting oxygen saturation ≥90% at the time of screening. Exclusion Criteria: 1. Any acute or severe comorbidities (e.g., severe infections or hematological, renal, metabolic, gastrointestinal, or endocrine dysfunction). 2. 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. 3. Life expectancy of less than 1 year. 4. Pregnant or breastfeeding women. 5. History of syncope or sustained ventricular tachyarrhythmia during exercise within the past 6 months. 6. Atrial fibrillation (AF). 7. Patients currently receiving or planning to receive treatment with disopyramide, cibenzoline, ranolazine, or a combination of beta-blockers with verapamil or diltiazem.

Outcomes

Primary Outcomes

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.

Time frame: From baseline to week 30.

Secondary Outcomes

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

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.

Time frame: At week 30.

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

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.

Time frame: At week 30.

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

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

Time frame: At week 30.

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

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

Time frame: From baseline to week 30.

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

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

Time frame: From baseline to week 30.

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

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

Time frame: From baseline to week 30.

Key safety endpoints

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

Time frame: From baseline to week 30.

Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes