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CompletedNCT04766892EMBARK-HFpEFUpdated Mar 20, 2025Results posted

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

A Phase 2 interventional study of mavacamten in Heart Failure With Preserved Ejection Fraction, sponsored by Bristol-Myers Squibb. Completed at 21 sites in 2 countries. Open to participants aged 50 Years and older. Per ClinicalTrials.gov, last updated 2025-03-20.

Sponsored by Bristol-Myers Squibb · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
30
Allocation
Not applicable
Ages
50 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Heart Failure With Preserved Ejection Fraction

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Keywords

  • HFpEF
  • NT-proBNP
  • Cardiac troponin T
03

In context

Heart Failure

5,701 studies on the registry are indexed under Heart Failure; 1,220 are open to participants now.

This study's enrollment of 30 is below the median of 72 across 3,736 interventional studies indexed under Heart Failure.

Browse Heart Failure studies →

Lead sponsor

Bristol-Myers Squibb is the lead sponsor of 1,538 studies on the registry; 116 are open to participants now.

Of its 429 completed or terminated interventional studies of FDA-regulated products, 223 (52%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
50 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
30 participants (actual)

Study arms

  • Experimental
    mavacamten (MYK-461)

    Drug: mavacamten

Interventions

  • Drugmavacamten

    mavacamten capsules

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

    Time frame: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)

  2. Number of Participants With Adverse Events of Special Interest (AESIs)

    Adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment. AEs of special interest include: Symptomatic overdose, teratogenicity, and LVEF ≤30%

    Time frame: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)

  3. Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)

    Treatment-emergent serious adverse event (TESAE) is defined as any untoward medical occurrence at any dose that: Results in death, Is immediately life-threatening (places the participant at immediate risk of death from the event as it occurred), Requires inpatient hospitalization or prolongation of existing hospitalization, Results in persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions, Results in a congenital abnormality or birth defect, Is an important medical event that may not result in death, be life-threatening, or require hospitalization, but may be considered an SAE when, based upon appropriate medical judgment, it may require medical or surgical intervention to prevent any of the outcomes listed above.

    Time frame: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)

  4. Number of Participants With Laboratory Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)

    Blood samples were collected to assess the abnormalities in laboratory parameters.

    Time frame: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)

  5. Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)

    Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

    Time frame: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)

  6. Number of Participants With Cardiac Rhythm Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)

    Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

    Time frame: From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)

  7. Ratio to Baseline at Week 26 in Resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) Levels

    Ratio to baseline in N-terminal pro B-type natriuretic peptide levels. The baseline value is defined as the last available value before the first administration of study drug.

    Time frame: At Week 26

  8. Ratio to Baseline at Week 26 in Resting High Sensitivity Cardiac Troponin T (Hs-cTnT) Levels Assessed by High-Sensitivity Assay

    Ratio to Baseline in resting high sensitivity cardiac troponin T (hs-cTnT) levels. The baseline value is defined as the last available value before the first administration of study drug.

    Time frame: At Week 26

07

Results

Posted Mar 20, 2025

Participant flow

Participant flow — Overall Study
MilestoneMavacamten
Started30
Safety population30
Intent to treat population30
Completed24
Not completed6
Withdrew: Withdrawal by subject1
Withdrew: Lost to follow-up1
Withdrew: Adverse event3
Withdrew: Left ventricular ejection fraction (lvef) criteria met1

Outcome measures

PrimaryNumber of Participants With Treatment-Emergent Adverse Events (TEAEs)

Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

Time frame:
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Reported as:
Count of participants · Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
ParticipantsMavacamten
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)23
PrimaryNumber of Participants With Adverse Events of Special Interest (AESIs)

Adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment. AEs of special interest include: Symptomatic overdose, teratogenicity, and LVEF ≤30%

Time frame:
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Reported as:
Count of participants · Participants
Number of Participants With Adverse Events of Special Interest (AESIs)
ParticipantsMavacamten
Symptomatic Overdose0
Outcomes of Pregnancy0
Left Ventricular Ejection Fraction (LVEF) ≤30%0
PrimaryNumber of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)

Treatment-emergent serious adverse event (TESAE) is defined as any untoward medical occurrence at any dose that: Results in death, Is immediately life-threatening (places the participant at immediate risk of death from the event as it occurred), Requires inpatient hospitalization or prolongation of existing hospitalization, Results in persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions, Results in a congenital abnormality or birth defect, Is an important medical event that may not result in death, be life-threatening, or require hospitalization, but may be considered an SAE when, based upon appropriate medical judgment, it may require medical or surgical intervention to prevent any of the outcomes listed above.

Time frame:
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Reported as:
Count of participants · Participants
Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
ParticipantsMavacamten
Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)5
PrimaryNumber of Participants With Laboratory Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)

Blood samples were collected to assess the abnormalities in laboratory parameters.

Time frame:
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Reported as:
Count of participants · Participants
Number of Participants With Laboratory Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)
ParticipantsMavacamten
Number of Participants With Laboratory Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)4
PrimaryNumber of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)

Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

Time frame:
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Reported as:
Count of participants · Participants
Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)
ParticipantsMavacamten
Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)4
PrimaryNumber of Participants With Cardiac Rhythm Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)

Treatment-emergent Adverse event (TEAE) is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

Time frame:
From the first dose of study drug through 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days)
Reported as:
Count of participants · Participants
Number of Participants With Cardiac Rhythm Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)
ParticipantsMavacamten
Number of Participants With Cardiac Rhythm Abnormalities Reported as Treatment-Emergent Adverse Events (TEAEs)5
PrimaryRatio to Baseline at Week 26 in Resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) Levels

Ratio to baseline in N-terminal pro B-type natriuretic peptide levels. The baseline value is defined as the last available value before the first administration of study drug.

Time frame:
At Week 26
Reported as:
Geometric mean · Ratio
Ratio to Baseline at Week 26 in Resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) Levels
RatioMavacamten
Ratio to Baseline at Week 26 in Resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) Levels0.7354 (0.5612 to 0.9637)
PrimaryRatio to Baseline at Week 26 in Resting High Sensitivity Cardiac Troponin T (Hs-cTnT) Levels Assessed by High-Sensitivity Assay

Ratio to Baseline in resting high sensitivity cardiac troponin T (hs-cTnT) levels. The baseline value is defined as the last available value before the first administration of study drug.

Time frame:
At Week 26
Reported as:
Geometric mean · Ratio
Ratio to Baseline at Week 26 in Resting High Sensitivity Cardiac Troponin T (Hs-cTnT) Levels Assessed by High-Sensitivity Assay
RatioMavacamten
Ratio to Baseline at Week 26 in Resting High Sensitivity Cardiac Troponin T (Hs-cTnT) Levels Assessed by High-Sensitivity Assay0.8645 (0.7711 to 0.9693)

Adverse events

Collected over Participants were assessed for All-Cause Mortality from first dose of study drug until their study completion (assessed up to approximately 1063 days) SAEs and Other AEs were assessed from first dose to 56 days following last dose of study drug (assessed for an average of 225 days up to a max of approximately 270 days). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Mavacamten1/30 (3.3%)5/30 (16.7%)18/30 (60%)
Most frequent serious events
Most frequent serious events
EventMavacamten
Atrial fibrillationCardiac disorders1/30
Cardiac failure acuteCardiac disorders1/30
Eye haemorrhageEye disorders1/30
GastroenteritisInfections and infestations1/30
DehydrationMetabolism and nutrition disorders1/30
Squamous cell carcinoma of skinNeoplasms benign, malignant and unspecified (incl cysts and polyps)1/30
Renal impairmentRenal and urinary disorders1/30
Most frequent other events
Most frequent other events
EventMavacamten
COVID-19Infections and infestations4/30
FallInjury, poisoning and procedural complications4/30
DizzinessNervous system disorders4/30
Upper respiratory tract infectionInfections and infestations3/30
HeadacheNervous system disorders3/30
DyspnoeaRespiratory, thoracic and mediastinal disorders3/30
HypertensionVascular disorders3/30
Atrial flutterCardiac disorders2/30
AstheniaGeneral disorders2/30
Urinary tract infectionInfections and infestations2/30

Baseline characteristics

Age, Continuous
Age, Continuous(Years)Mavacamten
Mean75.0 ± 7.58
Sex: Female, Male
Sex: Female, Male(Participants)Mavacamten
Female16
Male14
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Mavacamten
Hispanic or Latino7
Not Hispanic or Latino23
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Mavacamten
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American3
White26
More than one race0
Unknown or Not Reported1
08

Study locations

21 sites
  • Local Institution - 0028
    Birmingham, Alabama 35249, United States
  • Local Institution - 0019
    Phoenix, Arizona 85016, United States
  • Local Institution - 0011
    Tucson, Arizona 85724, United States
  • Local Institution - 0005
    Los Angeles, California 90027, United States
  • Local Institution - 0026
    San Francisco, California 94158, United States
  • Local Institution - 0020
    Jacksonville, Florida 32216, United States
  • Local Institution - 0014
    Miami, Florida 33133, United States
  • Local Institution - 0018
    Atlanta, Georgia 30322, United States
  • Local Institution - 0004
    Chicago, Illinois 60611, United States
  • Local Institution - 0023
    Hazel Crest, Illinois 60429, United States
  • Local Institution - 0017
    Slidell, Louisiana 70458, United States
  • Local Institution - 0007
    Grand Rapids, Michigan 49503, United States
  • Local Institution - 0012
    New York, New York 10065, United States
  • Local Institution - 0003
    Durham, North Carolina 27710, United States
  • Local Institution - 0016
    Oklahoma City, Oklahoma 73135, United States
  • Local Institution - 0010
    Portland, Oregon 97225, United States
  • Local Institution - 0001
    Portland, Oregon 97239, United States
  • Local Institution - 0002
    Philadelphia, Pennsylvania 19104, United States
  • Local Institution - 0008
    Charleston, South Carolina 29425, United States
  • Local Institution - 0006
    Salt Lake City, Utah 84112, United States
  • Local Institution - 0034
    Toronto, Ontario M5S 1B2, Canada
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Sep 1, 2021

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 20, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04766892
Lead sponsor
Bristol-Myers Squibb
Responsible party
Sponsor
First posted
Feb 23, 2021
Start date
Mar 30, 2021
Primary completion
Feb 26, 2024
Completion
Feb 26, 2024
Results posted
Mar 20, 2025
Last update
Mar 20, 2025

Study contacts

Bristol-Myers Squibb
study director · Bristol-Myers Squibb

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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