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A Study of Mavacamten in Participants With HFpEF and Chronic Elevation of cTnI and/or NT-proBNP (EMBARK-HFpEF)

  • Clinicaltrials.gov identifier

    NCT04766892

  • Recruitment Status

    Recruiting

  • First Posted

    February 23, 2021

  • Last update posted

    June 14, 2021

Study Description

Brief summary:

This is a Phase 2a proof-of-concept study to assess safety, tolerability, and preliminary efficacy of mavacamten treatment on biomarkers levels in participants with heart failure with preserved ejection fraction (HFpEF) and chronic elevation of cTnI and/or NT-proBNP. Data from this study will inform future study designs of mavacamten in participants with HFpEF.

  • Condition or Disease:Heart Failure With Preserved Ejection Fraction
  • Intervention/Treatment: Drug: Mavacamten
  • Phase: Phase 2

Detailed Description

N/A

Study Design

  • Study Type: Interventional
  • Estimated Enrollment: 35 participants
  • Intervention Model: Single Group Assignment
  • Masking: None (Open Label) ()
  • Primary Purpose: Treatment
  • Official Title: An Exploratory, Open-label, Proof-of-concept, Phase 2a Study of Mavacamten (MYK-461) in Participants With Heart Failure With Preserved Ejection Fraction (HFpEF) and Chronic Elevation of Cardiac Troponin I and/or NT-proBNP
  • Actual Study Start Date: March 2021
  • Estimated Primary Completion Date: May 2022
  • Estimated Study Completion Date: May 2022

Arms and interventions

Arm Intervention/treatment
Experimental: Mavacamten (MYK-461)
Drug: Mavacamten
Mavacamten Capsules

Outcome Measures

  • Primary Outcome Measures: 1. Frequency and severity of treatment-emergent adverse events, adverse events of special interest, and serious adverse events. [ Time Frame: 26 weeks ]
  • 2. Mavacamten effect on cTnI levels (at rest) [ Time Frame: 26 weeks ]
    Specifically, change from baseline to Week 26 in cTnI (resting), as assessed by a high-sensitivity assay
  • 3. Mavacamten effect on NT-proBNP levels (at rest) [ Time Frame: 26 weeks ]
    Specifically, change from baseline to Week 26 in NT-proBNP (resting)

Eligibility Criteria

  • Ages Eligible for Study: 50 Years and older (Adult, Older Adult)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No

Criteria

Key Inclusion Criteria: 1. Is at least 50 years old at Screening. 2. Body weight is greater than 45 kg at Screening. 3. Documented prior objective evidence of heart failure as shown by 1 or more of the following criteria: - Previous hospitalization for heart failure with documented radiographic evidence of pulmonary congestion. - Elevated LV end-diastolic pressure or pulmonary capillary wedge pressure at rest (≥15 mm Hg) or with exercise (≥25 mm Hg). - Elevated level of NT-proBNP (>400 pg/mL) or brain natriuretic peptide (BNP) (>200 pg/mL). - Echocardiographic evidence of medial E/e' ratio ≥ 15 or left atrial enlargement (left atrial volume index >34 mL/m2) together with chronic treatment with spironolactone, eplerenone, or a loop diuretic. 4. Meets 1 or more of the following criteria: - A hs-cTnI >99th percentile at screening OR - NT-proBNP* >300 pg/mL at initial screening measurement (if not in atrial fibrillation or atrial flutter) or >750 pg/mL (if in atrial fibrillation or atrial flutter). OR - If the screened participant is either of African descent or has a body mass index ≥30.0 kg/m2, a screening NT-proBNP* >240 pg/mL (if not in atrial fibrillation or atrial flutter) or >600 pg/mL (if in atrial fibrillation or atrial flutter). 5. Has documented LVEF ≥60% at the Screening visit and no history of prior LVEF ≤ 45% 6. Has documented elevated left ventricular mass index (LVMI) by 2-dimensional imaging (>95 g/m2 if female and >115 g/m2 if male) OR maximal left ventricular wall thickness ≥12 mm. 7. Has adequate acoustic windows on screening resting TTE 8. Has NYHA class II or III symptoms at Screening. Key Exclusion Criteria: 1. Has a prior diagnosis of HCM OR a known infiltrative or storage disorder causing HFpEF and/or cardiac hypertrophy, such as amyloidosis, Fabry disease, or Noonan syndrome with LV hypertrophy OR a positive serum immunofixation result. 2. Has a history of syncope within the last 6 months or sustained ventricular tachycardia with exercise within the past 6 months. 3. Has a history of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator discharge within 6 months prior to Screening. 4. Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or is not adequately rate controlled within 6 months prior to Screening. 5. Currently treated or planned treatment during the study with either: (a) a combination of beta blocker and verapamil or a combination of beta blocker and diltiazem, (b) disopyramide, or (c) biotin or biotin-containing supplements/multivitamins. 6. Has known moderate or severe aortic valve stenosis, hemodynamically significant mitral stenosis, or severe mitral or tricuspid regurgitation at Screening 7. Has severe chronic obstructive pulmonary disease, or other severe pulmonary disease, requiring home oxygen, chronic nebulizer therapy, chronic oral steroid therapy or hospitalized for pulmonary decompensation within 12 months. 8. Has body mass index ≥45.0 kg/m2 9. Has left ventricular global longitudinal strain by TTE in the range from 0 to -12.0 (assessed by central TTE reader) 10. Has NT-proBNP at Screening >2000 pg/mL

Contacts and Locations

Contacts

Contact: BMS Medical Information Team (Use email contact) clinical.trials@bms.com

Contact: Myokardia Medical Information Team Study Director

Locations

United States, California
Cedars-Sinai Medical Center
Los Angeles

United States, Florida
Jacksonville Center for Clinical Research
Jacksonville

United States, Florida
Infinite Clinical Research
Miami

United States, Illinois
Northwestern University
Chicago

United States, Louisiana
Louisiana Heart Center
Slidell

United States, Michigan
Spectrum Health
Grand Rapids

United States, New York
Weill Cornell Medical Center
New York

United States, North Carolina
Duke University Medical Center
Durham

United States, Ohio
The Lindner Center for Research and Education at the Christ Hospital
Cincinnati

United States, Oklahoma
South Oklahoma Heart Research
Oklahoma City

United States, Oregon
Providence St. Vincent Medical Center
Portland

United States, Oregon
Oregon Health & Science University
Portland

United States, Pennsylvania
University of Pennsylvania - Heart and Vascular Center
Philadelphia

United States, South Carolina
Medical University of South Carolina (MUSC)
Charleston

United States, Tennessee
Stern Cardiovascular Foundation, Inc
Germantown

United States, Utah
University of Utah Hospital & Clinics
Salt Lake City

Sponsors and Collaborators

MyoKardia, Inc.

More Information

  • Responsible Party: MyoKardia, Inc.
  • ClinicalTrials.gov Identifier: NCT04766892 History of Changes
  • Other Study ID Numbers: MYK-461-019
  • First Posted: February 23, 2021 Key Record Dates
  • Last Update Posted: June 14, 2021
  • Last Verified: June 2021
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Product Manufactured in and Exported from the U.S.: Yes
  • Keywords provided by MyoKardia, Inc.: NT-proBNP
    Cardiac troponin I
    HFpEF
  • Additional relevant MeSH terms: Heart Failure