A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy

NCT05414175 | Completed Phase 3 Results FDA Drug

• 1 other identifier: CV027-004
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 20

Brief Summary

The purpose of this study is to evaluate the effectiveness, safety, and tolerability of a 30-week course of mavacamten and the long-term effects of mavacamten in Japanese participants with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

Conditions

Cardiomyopathy, Hypertrophic Obstructive

Keywords

Mavacamten; Obstructive Hypertrophic Cardiomyopathy (HCM)

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30

  The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the doppler echocardiography. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.

  At Baseline and Week 30

Secondary Outcomes (5)

• Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30

  At Baseline and Week 30

• Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30

  Baseline and at Week 30

• Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30

  At baseline and week 30

• Change From Baseline in Cardiac Troponin I at Week 30

  At baseline and week 30

• Change From Baseline in Cardiac Troponin T at Week 30

  At baseline and week 30

Study Arms (1)

Mavacamten

EXPERIMENTAL

Drug: Mavacamten

Interventions

Mavacamten DRUG

Specified dose on specified days

Also known as: BMS-986427, MYK-461

Mavacamten

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 and greater, body weight ≥ 35kg
• Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
• Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation Society guidelines
• Has documented left ventricular ejection fraction (LVEF) ≥60% NYHA Class II or III

You may not qualify if:

• Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
• History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
• History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
• Paroxysmal atrial fibrillation with atrial fibrillation present at the time of Screening.
• Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
• Treatment (within 14 days prior to Screening) or planned treatment during the study with cibenzoline, disopyramide or ranolazine
• Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
• Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation \[ASA\]) within 6 months prior to Screening or plans to have either of these treatments during the study
• ICD placement within 2 months prior to Screening or planned ICD placement during the study
• Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation procedures, or completion
• Prior treatment with cardiotoxic agents such as doxorubicin or similar

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (20)

Local Institution - 0020

Uwajima, Ehime, 798-8510, Japan

Location

Local Institution - 0026

Himeji-Shi, Hyōgo, 672-8044, Japan

Location

Local Institution - 0016

Kobe, Hyōgo, 650-0047, Japan

Location

Local Institution - 0017

Tsukuba, Ibaraki, 305-0005, Japan

Location

Local Institution - 0023

Kanazawa, Ishikawa-ken, 920-8641, Japan

Location

Local Institution - 0019

Yokohama, Kanagawa, 227-8501, Japan

Location

Local Institution - 0015

Nankoku-shi, Kochi, 783-8505, Japan

Location

Local Institution - 0028

Tsu, Mie-ken, 514-8507, Japan

Location

Local Institution - 0027

Sendai, Miyagi, 980-8574, Japan

Location

Local Institution - 0014

Suita, Osaka, 564-8565, Japan

Location

Local Institution - 0011

Suita-Shi, Osaka, 565-0871, Japan

Location

Local Institution - 0012

Hamamatsu, Shizuoka, 431-3192, Japan

Location

Local Institution - 0010

Bunkyo-Ku, Tokyo, 113-0033, Japan

Location

Local Institution - 0009

Chuo-Ku, Tokyo, 104-0044, Japan

Location

Local Institution - 0003

Fuchu-Shi, Tokyo, 1830003, Japan

Location

Local Institution - 0001

Itabashi-Ku, Tokyo, 173-0003, Japan

Location

Local Institution - 0007

Koto-Ku, Tokyo, 135-0061, Japan

Location

Local Institution - 0005

Shinjuku-Ku, Tokyo, 160-8582, Japan

Location

Local Institution - 0013

Shinjuku-ku, Tokyo, 162-8666, Japan

Location

Local Institution - 0018

Osaka, 558-8558, Japan

Location

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

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

Results Point of Contact

• Title: Bristol-Myers Squibb Study Director
• Organization: Bristol-Myers Squibb

Clinical.Trials@bms.com

Please email

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2022
• First Posted: June 10, 2022
• Study Start: August 19, 2022
• Primary Completion: November 27, 2023
• Study Completion: December 11, 2025
• Last Updated: January 27, 2026
• Results First Posted: December 4, 2024

Record last verified: 2026-01

Locations

JP (20)