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Trial record 2 of 11 for:    mavacamten

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

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ClinicalTrials.gov Identifier: NCT05174416
Recruitment Status : Recruiting
First Posted : December 30, 2021
Last Update Posted : January 19, 2022
Sponsor:
Information provided by (Responsible Party):
LianBio LLC

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.

Condition or disease Intervention/treatment Phase
Obstructive Hypertrophic Cardiomyopathy Drug: Mavacamten Drug: Placebo Phase 3

Detailed Description:
This is a randomized, double-blinded, placebo-controlled clinical study witha long-term extension to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM. Approximately 81eligible participants will be enrolled and randomized in a 2:1 ratio (mavacamten:placebo). Participants will receive mavacamten or matching placebofor 30 weeks indouble-blinded manner. After 30-week double-blinded placebo-controlled treatment, eligible participants will receive mavacamten for additional 48 weeks (placebogroup: switch from placebo to mavacamten, mavacamten group: maintain on mavacamten).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 81 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase III, Randomized, Double-blinded, Placebo-controlled Clinical Study With A Long-term Extension to Evaluate the Efficacy and Safety of Mavacamten in Chinese Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Actual Study Start Date : January 4, 2022
Estimated Primary Completion Date : November 2022
Estimated Study Completion Date : May 2024


Arm Intervention/treatment
Experimental: Mavacamten
Mavacamten Capsules
Drug: Mavacamten
Mavacamten Capsules
Other Name: MYK-461

Placebo Comparator: placebo
Matching Placebo Capsules
Drug: Placebo
Matching PBO capsules during placebo controlled period,and mavacamten capsules during long term extension period




Primary Outcome Measures :
  1. Change from baseline to Week 30 in Valsalva LVOT peak gradient [ Time Frame: 30 weeks ]
    To compare the effect of a 30-week course of mavacamten with placebo on Valsalva left ventricular outflow tract (LVOT) peak gradient as determined by Doppler echocardiography


Secondary Outcome Measures :
  1. Change from baseline to Week 30 in resting LVOT peak gradient Proportion of participants achieving a Valsalva LVOT peak gradient < 30 mmHg at Week 30 Proportion of participants achieving a Valsalva LVOT peak gradient < 50 mmHg at Week 30 [ Time Frame: 30 weeks ]

    To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction.

    To compare the effect of a 30-week course of mavacamten with placebo on clinical symptoms To compare the effect of a 30-week course of mavacamten with placebo on Participant-Reported health status individually To compare the effect of a 30-week course of mavacamten on cardiac biomarkers To compare the effect of a 30-week course of mavacamten with placebo on left ventricular (LV) mass evaluated by cardiac magnetic resonance (CMR) imaging


  2. Proportion of participants with at least 1 class improvement in NYHA functional classification from baseline to Week 30 [ Time Frame: 30 weeks ]

    To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction.

    To compare the effect of a 30-week course of mavacamten with placebo on clinical symptoms To compare the effect of a 30-week course of mavacamten with placebo on Participant-Reported health status individually To compare the effect of a 30-week course of mavacamten on cardiac biomarkers To compare the effect of a 30-week course of mavacamten with placebo on left ventricular (LV) mass evaluated by cardiac magnetic resonance (CMR) imaging


  3. Change from baseline to Week 30 in KCCQ Clinical Summary Score (CSS) Change from baseline to Week 30 in LV mass index Change from baseline to Week 30 in NT-proBNP Change from baseline to Week 30 in cardiac troponin [ Time Frame: 30 weeks ]

    To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction.

    To compare the effect of a 30-week course of mavacamten with placebo on clinical symptoms To compare the effect of a 30-week course of mavacamten with placebo on Participant-Reported health status individually To compare the effect of a 30-week course of mavacamten on cardiac biomarkers To compare the effect of a 30-week course of mavacamten with placebo on left ventricular (LV) mass evaluated by cardiac magnetic resonance (CMR) imaging



Other Outcome Measures:
  1. Proportion of participants achieving NYHA Class I and resting and Valsalva LVOT peak gradient < 30 mmHg at Week 30 Change from baseline to Week 30 in echocardiographic indices of cardiac structure and systolic and diastolic function [ Time Frame: 30 weeks ]
    To assess the effect of a 30-week course of mavacamten on cardiac function and structure as evaluated by echocardiography To assess the effect of a 30-week course of mavacamten on cardiac function and structure as evaluated by CMR imaging To assess the effect of a 30-week course of mavacamten on Participant-Reported health status

  2. Change from baseline to Week 30 in myocardial fibrosis Change from baseline to Week 30 in cellular hypertrophy, cardiac structure, and function Change from baseline to Week 30 in Total Symptom Score and Overall Summary Score from KCCQ [ Time Frame: 30 weeks ]
    To assess the effect of a 30-week course of mavacamten on cardiac function and structure as evaluated by echocardiography To assess the effect of a 30-week course of mavacamten on cardiac function and structure as evaluated by CMR imaging To assess the effect of a 30-week course of mavacamten on Participant-Reported health status

  3. Incidence of LVEF < 50% determined by TTE Incidence and severity of TEAEs, and treatment-emergent SAEs [ Time Frame: 30 weeks ]
    To assess the safety of mavacamten during the 30-week double-blinded, placebo-controlled treatment period

  4. Incidence of major adverse cardiac events (MACEs; CV death, non-fatal stroke, non-fatal myocardial infarction) Incidence of hospitalizations (due to CV and non-CV events) [ Time Frame: 30 weeks ]
    To assess the safety of mavacamten during the 30-week double-blinded, placebo-controlled treatment period

  5. Incidence of HF events, including hospitalizations and urgent emergency room/outpatient visits for HF Incidence of atrial fibrillation/flutter (new from screening, and recurrent) Incidence of ICD therapy and resuscitated cardiac arrest [ Time Frame: 30 weeks ]
    To assess the safety of mavacamten during the 30-week double-blinded, placebo-controlled treatment period

  6. Incidence of ventricular tachyarrhythmias including ventricular tachycardia, ventricular fibrillation, and Torsades de Pointe Incidence of adverse events of special interest (AESIs; symptomatic overdose, outcomes of pregnancy, LVEF ≤ 30%) [ Time Frame: 30 weeks ]
    To assess the safety of mavacamten during the 30-week double-blinded, placebo-controlled treatment period



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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
  • 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.

Exclusion Criteria:

  • 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, with the exception of hepatitis B s-antibody positive, which is a marker of immunity.
  • Known infection with COVID-19 (coronavirus disease 2019) within 90 days of 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
  • Unable to comply with the study requirements, including the number of required visits to the clinical site
  • Is a first degree relative of personnel directly affiliated with the study at the clinical study site, any study vendor, or the study sponsor.
  • Identified as alcohol addicts.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05174416


Contacts
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Contact: Wen Zhou, Bachelor 13811626643 Wen.Zhou@lianbio.com

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Shuyang Zhang    Shuyang    shuyangzhang103@163.com   
Sponsors and Collaborators
LianBio LLC
Investigators
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Principal Investigator: Shuyang Zhang, Doctor Peking Union Medical College Hospital
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Responsible Party: LianBio LLC
ClinicalTrials.gov Identifier: NCT05174416    
Other Study ID Numbers: LB2001-301
First Posted: December 30, 2021    Key Record Dates
Last Update Posted: January 19, 2022
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by LianBio LLC:
Mavacamten
Additional relevant MeSH terms:
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Cardiomyopathies
Cardiomyopathy, Hypertrophic
Hypertrophy
Heart Diseases
Cardiovascular Diseases
Pathological Conditions, Anatomical
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Aortic Valve Disease
Heart Valve Diseases