A Study to Evaluate the Efficacy and Safety of Mavacamten in Chinese Adults With Symptomatic Obstructive HCM

NCT05174416 | Completed Phase 3 Results FDA Drug

• 1 other identifier: LB2001-301
• Study Type: interventional
• Target: 81
• Locations: 1 country
• Sites: 1

Brief Summary

Mavacamtenis a novel, small molecule, selective allosteric inhibitor of cardiac-specific myosin, for the treatment of patients with symptomatic oHCM. This study will assess the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM.

Conditions

Obstructive Hypertrophic Cardiomyopathy

Keywords

Mavacamten

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to Week 30 in Valsalva Left Ventricular Outflow Tract (LVOT) Peak Gradient

  To compare the effect of a 30-week course of mavacamten with placebo on Valsalva LVOT peak gradient as determined by Doppler echocardiography

  30 weeks

Secondary Outcomes (8)

• Change From Baseline to Week 30 in Resting LVOT Peak Gradient

  30 weeks

• Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 30 mmHg at Week 30

  30 weeks

• Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 50 mmHg at Week 30.

  30 weeks

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

  30 weeks

• Change From Baseline to Week 30 in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS)

  30 weeks

Study Arms (2)

Mavacamten

EXPERIMENTAL

Mavacamten Capsules

Drug: Mavacamten

placebo

PLACEBO COMPARATOR

Matching Placebo Capsules

Drug: Placebo

Interventions

Mavacamten DRUG

Mavacamten Capsules

Also known as: MYK-461, BMS-986327

Mavacamten

Placebo DRUG

Matching PBO capsules during placebo controlled period，and mavacamten capsules during long term extension period

placebo

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Is at least 18 years old at screening.
• Body weight is greater than 45 kg at screening.
• Has adequate acoustic windows to enable accurate TTEs
• Diagnosed with oHCM
• Has documented LVEF ≥ 55% at rest.
• Has a valid measurement of Valsalva LVOT peak gradient at screening
• Has NYHA Class II or III symptoms at screening
• Female participants must not be pregnant or lactating
• Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to national, local, and institutional guidelines before the first study specific procedure.

You may not qualify if:

• Participated in a clinical trial in which the participant received any investigational drug (or is currently using an investigational device) within 30 days prior to screening, or at least 5 times the respective elimination half-life (if known), whichever is longer.
• Causing cardiac hypertrophy in other reasons
• Previously participated in a clinical study with mavacamten.
• Hypersensitivity to any of the components of the mavacamten formulation.
• Current treatment (within 14 days prior to screening) or planned treatment during the double-blinded treatment with a combination of beta-blockers and verapamil or a combination of beta-blockers and diltiazem.
• Has been successfully treated with invasive septal reduction
• Has documented obstructive coronary artery disease
• Has known moderate or severe (as per investigator's judgment) aortic valve stenosis, constrictive pericarditis, or clinically significant congenital heart disease at screening.
• Has any acute or serious comorbid condition that, in the judgment of the investigator, could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study.
• History of malignant disease within 10 years of screening
• Has safety laboratory parameters outside normal limits at screening as assessed by the local laboratory
• Has a positive serologic test at screening for infection with human immunodeficiency virus, hepatitis C virus, or hepatitis B virus surface antigen.
• Known uncured COVID-19 (coronavirus disease 2019) infection or with severe complication before screening.
• 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 cardio toxic agents.

Sponsors & Collaborators

• LianBio LLC: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Related Publications (4)

• Tian Z, Li L, Li X, Zhang Q, Peng D, Ma W, Yang P, Wang F, Cheng X, Fu Y, Sun J, Wang J, Zhang S. Effects of Mavacamten on Cardiac Magnetic Resonance Features in Chinese Patients With Obstructive Hypertrophic Cardiomyopathy. JACC Asia. 2025 Aug;5(8):1064-1074. doi: 10.1016/j.jacasi.2025.05.015. Epub 2025 Jul 8.

  PMID: 40632050 DERIVED

• Tian Z, Li X, Li L, Zhang Q, Wang J, Shi Y, Peng D, Yang P, Ma W, Wang F, Jin W, Cheng X, Chen YM, Zhong Y, Barrett YC, Zheng J, Zhang S. Effect of Mavacamten on Echocardiographic Features in Chinese Patients with Obstructive Hypertrophic Cardiomyopathy: Results from the EXPLORER-CN Study. Cardiol Ther. 2025 Jun;14(2):267-282. doi: 10.1007/s40119-025-00409-5. Epub 2025 Apr 29.

  PMID: 40299193 DERIVED

• Tian Z, Li L, Li X, Wang J, Zhang Q, Li Z, Peng D, Yang P, Ma W, Wang F, Jin W, Cheng X, Sun J, Fu Y, Lyu C, Zhang S. Effect of Mavacamten on Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy: The EXPLORER-CN Randomized Clinical Trial. JAMA Cardiol. 2023 Oct 1;8(10):957-965. doi: 10.1001/jamacardio.2023.3030.

  PMID: 37639259 DERIVED

• Tian Z, Wang F, Jin W, Zhang Q, Zhou J, Yang P, Wang G, Hsu P, Sun J, Zhang S, Han Y. Study design and rationale of EXPLORER-CN: a phase III, randomised, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy. BMJ Open. 2023 Jun 19;13(6):e071473. doi: 10.1136/bmjopen-2022-071473.

  PMID: 37336533 DERIVED

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: Prof. Shuyang Zhang
• Organization: Peking Union Medical College Hospital

shuyangzhang103@163.com

+86 13911667211

Study Officials

• Shuyang Zhang, M.D., Ph.D.

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2021
• First Posted: December 30, 2021
• Study Start: January 4, 2022
• Primary Completion: March 6, 2023
• Study Completion: July 22, 2024
• Last Updated: September 19, 2024
• Results First Posted: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)