Background and Aims: Obesity and type 2 diabetes (T2D) are highly prevalent in people who have heart failure (HF) with preserved ejection fraction (HFpEF) and are associated with an especially high burden of symptoms and functional impairment. No approved therapies specifically target obesity-related HFpEF in patients with T2D. The STEP-HFpEF DM randomised controlled trial (NCT04916470) tested the efficacy and safety of semaglutide 2.4 mg compared with placebo in patients with obesity-related HFpEF and T2D.
Methods: In this double-blind trial, we randomised 616 patients across 108 sites in 16 countries with HFpEF, a body mass index (BMI) of ≥30 kg/m2 and T2D to once-weekly semaglutide 2.4 mg or placebo for 52 weeks. Dual primary endpoints were change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) and body weight. Confirmatory secondary endpoints were change in 6-minute walk distance (6MWD), hierarchical composite (death, HF events, and change in KCCQ-CSS and 6MWD) and change in C-reactive protein. Exploratory endpoints included NTproBNP and adjudicated HF events.
Results: Median age was 69 years, 44% were women, median BMI was 36.9 kg/m2, KCCQ-CSS was 59.4 points, 6MWD was 280 meters, N-terminal pro B-type natriuretic peptide was 493 pg/mL, left ventricular ejection fraction was 56%, and 29.4% were in New York Heart Association functional (NYHA) class II–IV. Most patients were treated with diuretics (80.8%); 32.5% received mineralocorticoid receptor antagonists and 32.8% received sodium–glucose co‐transporter-2 inhibitors.
Conclusion: STEP-HFpEF DM randomised patients with obesity-related HFpEF and T2D who have marked symptomatic and functional impairment, and will determine whether semaglutide can improve symptoms, physical limitations and exercise function in this group with a large need for additional therapies.