What the trial was testing
The EMBARK-HFpEF enrolled 30 patients with heart failure. The study was sponsored by Bristol-Myers Squibb and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
42% of patients showed improvement in heart failure symptoms with mavacamten treatment.
JAMA cardiology · 2025 · NCT04766892
These findings — that nearly half of patients showed improvement in heart failure symptoms and exercise capacity — were published in the JAMA cardiology and represent the headline result of the study.
Researchers tracked outcomes across 30 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with heart failure, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This experimental drug showed signs of helping people with a type of heart failure that doesn't have many good treatment options. While the results are encouraging, this is still early-stage testing in a small group. Talk to your cardiologist about whether you might qualify for ongoing trials or other available treatments.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open heart failure trials
Biomarker Guided Discharge of Heart Failure Patients
This is a multi-centre, single blind, randomized study. Patients admitted to hospital with acute decompensated heart failure will be randomized to biomarker guided discharge algorithm vs usual care in a 2:1 ratio. NTproBNP and other biomarkers will be measured within 24 hours of admission. The NTproBNP results will be used to further stratify participants randomized to the biomarker guided group into lower and medium to higher risk pathways. Biomarkers will be repeated after 2-3 days and again prior to discharge. Specific care pathways will be followed for each of the lower risk and medium-higher risk groups. Biomarkers will be repeated 30 days post discharge. Participants will be followed with a phone call at 3 months and return for a follow up visit at 6 months post discharge for outcome evaluation.
AMBulatory UltraSound for Heart Failure Management
AMBUSH study is a multicenter randomized, controlled, open-label clinical trial (PROBE (Prospective Randomized Open Blinded End-point) type). The main objective of AMBUSH study is to assess the effect of therapeutic management guided by pulmonary ultrasound and the assessment of the inferior vena cava in patients with heart failure seen in an ambulatory (outpatient) setting on a mixed clinical-biological endpoint (including variations of natriuretic peptides - NtProBNP) at 30 days.