Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy

Summary

This is a multicenter, randomized, placebo-controlled, 2-part study to evaluate the safety and efficacy of AT-001 in adult patients (N=675) with Diabetic Cardiomyopathy at high risk of progression to overt heart failure.

Timeline

Start

2019-09-20

Primary completion

2023-12

Completion

2025-12

Outcome

Missed primary endpoint

paper Among individuals with DbCM and impaired exercise capacity, treatment with AT-001 for 15 months did not result in significantly better exercise capacity compared with placebo. PMID 38597864 ↗

Publications

• Blumer V, Januzzi JL Jr, Liu Y, Butler J, Ezekowitz JA, Perfetti R, Rosenstock J, Del Prato S, Tang WHW, Urbinati A, Zannad F, Lewis GD, Solomon SD, Hedge S, Ibrahim NE, Lam CSP. Sex Differences in Diabetic Cardiomyopathy and Treatment Response to AT-001: Insights From the ARISE-HF Study. JACC Heart Fail. 2026 Jan;14(1):102433. doi: 10.1016/j.jchf.2025.02.015. Epub 2025 May 8.
• Siddiqi TJ, Liu Y, Zannad F, Tang WHW, Solomon S, Rosenstock J, Perfetti R, Marwick TH, Lewis GD, Lam CSP, Ibrahim NE, Ezekowitz J, Del Prato S, Butler J, Januzzi JL. Health status in stage B heart failure from diabetic cardiomyopathy baseline results from ARISE-HF. J Diabetes Complications. 2025 Jul;39(7):109059. doi: 10.1016/j.jdiacomp.2025.109059. Epub 2025 Apr 25.
• Tang WHW, Liu Y, Butler J, Del Prato S, Ezekowitz JA, Ibrahim NE, Lam CSP, Marwick TH, Perfetti R, Rosenstock J, Solomon SD, Zannad F, Januzzi JL, Lewis GD. Impaired Exercise Capacity in High-Risk Diabetic Cardiomyopathy: The ARISE-HF Cardiopulmonary Exercise Testing Subanalysis. Circ Heart Fail. 2025 Mar;18(3):e012200. doi: 10.1161/CIRCHEARTFAILURE.124.012200. Epub 2025 Jan 30.
• Marwick TH, Lam C, Liu Y, Del Prato S, Rosenstock J, Butler J, Ezekowitz J, Ibrahim NE, Tang WHW, Zannad F, Perfetti R, Januzzi JL. Echocardiographic phenotypes of diabetic myocardial disorder: evolution over 15 months follow-up in the ARISE-HF trial. Cardiovasc Diabetol. 2025 Jan 13;24(1):16. doi: 10.1186/s12933-024-02554-y.
• Gouda P, Liu Y, Butler J, Del Prato S, Ibrahim NE, Lam CSP, Marwick T, Rosenstock J, Tang W, Zannad F, Januzzi J, Ezekowitz J. Relationship between NT-proBNP, echocardiographic abnormalities and functional status in patients with subclinical siabetic cardiomyopathy. Cardiovasc Diabetol. 2024 Aug 2;23(1):281. doi: 10.1186/s12933-024-02378-w.
• Batista JL, Liu Y, Butler J, Del Prato S, Ezekowitz JA, Lam CSP, Marwick TH, Rosenstock J, Tang WHW, Perfetti R, Urbinati A, Zannad F, Januzzi JL Jr, Ibrahim NE. Racial Differences in Diabetic Cardiomyopathy: The ARISE-HF Trial. J Am Coll Cardiol. 2024 Jul 16;84(3):233-243. doi: 10.1016/j.jacc.2024.04.053.
• Januzzi JL Jr, Butler J, Del Prato S, Ezekowitz JA, Ibrahim NE, Lam CSP, Lewis GD, Marwick TH, Perfetti R, Rosenstock J, Solomon SD, Tang WHW, Zannad F. Randomized Trial of a Selective Aldose Reductase Inhibitor in Patients With Diabetic Cardiomyopathy. J Am Coll Cardiol. 2024 Jul 9;84(2):137-148. doi: 10.1016/j.jacc.2024.03.380. Epub 2024 Apr 8.
• Januzzi JL, Del Prato S, Rosenstock J, Butler J, Ezekowitz J, Ibrahim NE, Lam CSP, Marwick T, Wilson Tang WH, Liu Y, Mohebi R, Urbinati A, Zannad F, Perfetti R. Characterizing diabetic cardiomyopathy: baseline results from the ARISE-HF trial. Cardiovasc Diabetol. 2024 Feb 1;23(1):49. doi: 10.1186/s12933-024-02135-z.

Drugs

Indications