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A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT (EMBARK-HFpEF)

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ClinicalTrials.gov Identifier: NCT04766892
Recruitment Status : Recruiting
First Posted : February 23, 2021
Last Update Posted : February 9, 2022
Sponsor:
Information provided by (Responsible Party):
MyoKardia, Inc.

Brief Summary:
This is a Phase 2a proof-of-concept study to assess safety, tolerability, and preliminary efficacy of mavacamten treatment on biomarker levels in participants with heart failure with preserved ejection fraction (HFpEF) and elevation of NT-proBNP with or without elevation of cTnT. Data from this study will inform future study designs of mavacamten in patients with HFpEF.

Condition or disease Intervention/treatment Phase
Heart Failure With Preserved Ejection Fraction Drug: mavacamten Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 participants
Allocation: N/A
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 Biomarkers
Actual Study Start Date : March 30, 2021
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : March 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Failure

Arm Intervention/treatment
Experimental: mavacamten (MYK-461) Drug: mavacamten
mavacamten capsules




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 NT-proBNP levels (at rest) [ Time Frame: 26 weeks ]
    Specifically, change from baseline to Week 26 in NT-proBNP (resting)

  3. Mavacamten effect on cTnT levels (at rest) [ Time Frame: 26 weeks ]
    Specifically, change from baseline to Week 26 in cTnT (resting), as assessed by a high-sensitivity assay



Information from the National Library of Medicine

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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:

    1. A screening hs-cTnT ≥ 99th percentile AND a screening NT-proBNP > 200 pg/mL (if not in atrial fibrillation or atrial flutter) or > 500 pg/mL (if in atrial fibrillation or atrial flutter) OR if the screened participant is of African descent or has a body mass index (BMI) ≥ 30.0 kg/m2, a screening hs-cTnT ≥ 99th percentile, AND a screening NT-proBNP > 160 pg/mL (if not in atrial fibrillation or atrial flutter) or > 400 pg/mL (if in atrial fibrillation or atrial flutter).
    2. A screening NT-proBNP > 300 pg/mL (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 of African descent or has a BMI ≥ 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 maximal left ventricular wall thickness ≥12 mm OR documented elevated left ventricular mass index by 2-dimensional imaging (>95 g/m2 if female and >115 g/m2 if male).
  7. Has high quality TTEs without or with echocardiographic contrast agents.
  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 the central laboratory).
  10. Has NT-proBNP at Screening >2000 pg/mL.
  11. Has acute decompensated heart failure events requiring intravenous (IV) diuretics, IV inotropes, IV vasodilators, or a left ventricular assist device within 30 days prior to Screening.

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): NCT04766892


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

Locations
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Sponsors and Collaborators
MyoKardia, Inc.
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Responsible Party: MyoKardia, Inc.
ClinicalTrials.gov Identifier: NCT04766892    
Other Study ID Numbers: MYK-461-019
First Posted: February 23, 2021    Key Record Dates
Last Update Posted: February 9, 2022
Last Verified: January 2022

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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.: No
Keywords provided by MyoKardia, Inc.:
HFpEF
NT-proBNP
Cardiac troponin T
Additional relevant MeSH terms:
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Heart Failure
Heart Diseases
Cardiovascular Diseases