Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy

NCT07529938 | Completed

• 1 other identifier: FW-CHC-HCM-2024-01
• Study Type: observational
• Target: 222
• Locations: 1 country
• Sites: 1

Brief Summary

This retrospective cohort study aims to evaluate the clinical efficacy and safety of mavacamten in adult patients with obstructive hypertrophic cardiomyopathy (oHCM). A total of 222 patients were included and categorized based on treatments received in routine clinical practice into a mavacamten group and a standard therapy group. The primary outcome is the change in resting left ventricular outflow tract (LVOT) gradient at Week 30. Secondary outcomes include changes in Valsalva LVOT gradient, New York Heart Association (NYHA) functional class, cardiac biomarkers, and echocardiographic parameters. Safety outcomes include adverse events and left ventricular systolic dysfunction. This study provides real-world evidence on the effectiveness and safety of mavacamten in Chinese patients with oHCM.

Conditions

Hypertrophic Cardiomyopathy (HCM); Obstructive Hypertrophic Cardiomyopathy (oHCM)

Keywords

Mavacamten; Hypertrophic Cardiomyopathy; LVOT obstruction; Cardiac function; Real-world study; Retrospective cohort; Echocardiography

Outcome Measures

Primary Outcomes (1)

• Change in Resting Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30

  Change from baseline in resting left ventricular outflow tract (LVOT) peak gradient measured by Doppler echocardiography (unit: mmHg). Higher values indicate greater obstruction.

  Baseline to Week 30

Secondary Outcomes (10)

• Change in Valsalva-Induced Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30

  Baseline to Week 30

• Proportion of Participants with Improvement in New York Heart Association (NYHA) Functional Classification at Week 30

  Baseline to Week 30

• Change in N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) Levels at Week 30

  Baseline to Week 30

• Change in High-Sensitivity Cardiac Troponin I (hs-cTnI) Levels at Week 30

  Baseline to Week 30

• Change in Left Ventricular Ejection Fraction (LVEF) at Week 30

  Baseline to Week 30

Study Arms (2)

Mavacamten Group

Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received mavacamten as part of routine clinical practice. Patients were not assigned to interventions but were categorized based on treatment received.

Drug: mavacamten

Standard Therapy Group

Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received standard pharmacological therapy, including beta-blockers or non-dihydropyridine calcium channel blockers, in routine clinical practice.

Interventions

mavacamten DRUG

Mavacamten was administered as part of routine clinical care for patients with obstructive hypertrophic cardiomyopathy. This observational study did not assign interventions; instead, patients were categorized based on treatments received in real-world clinical practice. The initial dose was 2.5 mg once daily, with dose adjustments based on left ventricular ejection fraction and LVOT gradient as clinically indicated.

Also known as: Camzyos, Mai Fan Tuo

Mavacamten Group

Eligibility Criteria

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

Study Population

Adult patients diagnosed with obstructive hypertrophic cardiomyopathy who received treatment at Fuwai Central China Cardiovascular Hospital between January 2024 and May 2025 in routine clinical practice.

You may qualify if:

• Age ≥18 years
• Diagnosis of hypertrophic cardiomyopathy
• NYHA functional class II-III
• First-time treatment with mavacamten

You may not qualify if:

• Known hypersensitivity to mavacamten or its components
• Left ventricular ejection fraction (LVEF) \<55%
• Septal reduction therapy within 6 months prior to enrollment
• Presence of other conditions requiring long-term anticoagulation
• Pregnancy or breastfeeding
• Any condition deemed unsuitable by the investigator

Sponsors & Collaborators

• Chinese Academy of Medical Sciences, Fuwai Hospital: lead

Study Sites (1)

Fuwai Central China Cardiovascular Hospital

Zhengzhou, Henan, 450000, China

Location

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic; Ventricular Outflow Obstruction, Left

Interventions

MYK-461

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases; Ventricular Outflow Obstruction

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Cardiologist

Study Record Dates

• First Submitted: April 8, 2026
• First Posted: April 14, 2026
• Study Start: January 1, 2024
• Primary Completion: May 30, 2025
• Study Completion: June 30, 2025
• Last Updated: April 17, 2026

Record last verified: 2026-04

Locations

CN (1)