Status:

COMPLETED

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

Lead Sponsor:

Bristol-Myers Squibb

Conditions:

Heart Failure With Preserved Ejection Fraction

Eligibility:

All Genders

50+ years

Phase:

PHASE2

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 ejectio...

Eligibility Criteria

Inclusion

  • Key
  • Is at least 50 years old at Screening.
  • Body weight is greater than 45 kg at Screening.
  • 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.
  • Meets 1 or more of the following criteria:
  • 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).
  • 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).
  • Has documented LVEF ≥60% at the Screening visit and no history of prior LVEF ≤ 45%.
  • 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).
  • Has high quality TTEs without or with echocardiographic contrast agents.
  • Has NYHA class II or III symptoms at Screening.
  • Key

Exclusion

  • 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.
  • Has a history of syncope within the last 6 months or sustained ventricular tachycardia with exercise within the past 6 months.
  • Has a history of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator discharge within 6 months prior to Screening.
  • 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.
  • 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.
  • Has known moderate or severe aortic valve stenosis, hemodynamically significant mitral stenosis, or severe mitral or tricuspid regurgitation at Screening.
  • 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.
  • Has body mass index ≥45.0 kg/m2.
  • Has left ventricular global longitudinal strain by TTE in the range from 0 to -12.0 (assessed by the central laboratory).
  • Has NT-proBNP at Screening \>2000 pg/mL.
  • 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.

Key Trial Info

Start Date :

March 30 2021

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

February 26 2024

Estimated Enrollment :

30 Patients enrolled

Trial Details

Trial ID

NCT04766892

Start Date

March 30 2021

End Date

February 26 2024

Last Update

March 20 2025

Active Locations (21)

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Page 1 of 6 (21 locations)

1

Local Institution - 0028

Birmingham, Alabama, United States, 35249

2

Local Institution - 0019

Phoenix, Arizona, United States, 85016

3

Local Institution - 0011

Tucson, Arizona, United States, 85724

4

Local Institution - 0005

Los Angeles, California, United States, 90027

A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT | DecenTrialz