Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy

NCT ID: NCT06947590

Last Updated: 2025-04-27

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 78 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-04-20
• Study Completion Date: 2026-02-01

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Mavacamten Group

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.

Group Type: EXPERIMENTAL

mavacamten

Intervention Type: 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.

Control Group

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.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

mavacamten

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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Xu Liu

OTHER

Sponsor Role: lead

Responsible Party

Xu Liu

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

Shanghai Chest Hospital

Shanghai, , China

Countries

China

Other Identifiers

MVLOHCM

Identifier Type: -

Identifier Source: org_study_id