A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

Summary

This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP), Open-label Phase (OLP), and Post-Treatment Follow-up Period (PTFP). It is planned to randomize approximately 150 subjects at a 2:1 ratio of luspatercept versus placebo.

Timeline

Start

2018-02-05

Primary completion

2022-11-28

Completion

2022-11-28

Publications

• Background Piga A, Perrotta S, Gamberini MR, Voskaridou E, Melpignano A, Filosa A, Caruso V, Pietrangelo A, Longo F, Tartaglione I, Borgna-Pignatti C, Zhang X, Laadem A, Sherman ML, Attie KM. Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with beta-thalassemia. Blood. 2019 Mar 21;133(12):1279-1289. doi: 10.1182/blood-2018-10-879247. Epub 2019 Jan 7.
• Musallam KM, Cappellini MD, Kattamis A, Dyer M, Hnoosh A, Eliason L, Pelligra CG, Guo S, Mu Y, Medlin LF, Reverte M, Kuo KHM, Taher AT. Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent beta-Thalassemia Treated in the BEYOND Trial. Eur J Haematol. 2026 Jul;117(1):161-173. doi: 10.1111/ejh.70161. Epub 2026 Mar 19.
• Taher AT, Viprakasit V, Kattamis A, Perrotta S, Ricchi P, Porter JB, Coates TD, Forni GL, Musallam KM, Esposito O, Pilot R, Kuo WL, Lai Y, Reverte M, Wei R, Bueno LM, Cappellini MD. Efficacy and safety of luspatercept in non-transfusion-dependent beta-thalassemia: long-term results from the BEYOND study. Blood Adv. 2025 Dec 9;9(23):6108-6119. doi: 10.1182/bloodadvances.2025016554.
• Denton CC, Vodala S, Veluswamy S, Hofstra TC, Coates TD, Wood JC. Splenic iron decreases without change in volume or liver parameters during luspatercept therapy. Blood. 2023 Nov 30;142(22):1932-1934. doi: 10.1182/blood.2023021839.
• Taher AT, Musallam KM, Viprakasit V, Kattamis A, Lord-Bessen J, Yucel A, Guo S, Pelligra C, Shields AL, Shetty JK, Miteva D, Bueno LM, Cappellini MD. Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia. BMJ Open. 2023 Mar 22;13(3):e066683. doi: 10.1136/bmjopen-2022-066683.
• Taher AT, Cappellini MD, Kattamis A, Voskaridou E, Perrotta S, Piga AG, Filosa A, Porter JB, Coates TD, Forni GL, Thompson AA, Tartaglione I, Musallam KM, Backstrom JT, Esposito O, Giuseppi AC, Kuo WL, Miteva D, Lord-Bessen J, Yucel A, Zinger T, Shetty JK, Viprakasit V; BEYOND Investigators. Luspatercept for the treatment of anaemia in non-transfusion-dependent beta-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial. Lancet Haematol. 2022 Oct;9(10):e733-e744. doi: 10.1016/S2352-3026(22)00208-3. Epub 2022 Aug 22.

Drugs

Indications