drugset / Trial / NCT01571635

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.

Phase 2 Terminated 46 enrolled Celgene Corporation

Non-randomizedParallel-groupOpen-labelTreatment

Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia

Background Cappellini MD, Porter J, Origa R, Forni GL, Voskaridou E, Galacteros F, Taher AT, Arlet JB, Ribeil JA, Garbowski M, Graziadei G, Brouzes C, Semeraro M, Laadem A, Miteva D, Zou J, Sung V, Zinger T, Attie KM, Hermine O. Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase II, open-label, dose-finding study. Haematologica. 2019 Mar;104(3):477-484. doi: 10.3324/haematol.2018.198887. Epub 2018 Oct 18.
Background Cappellini M, et al. A Phase 2a, Open-Label, Dose-Finding Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta ( )-Thalassemia: Interim Results. Presented at the 55th Annual Meeting of the American Society of Hematology (ASH), December 7-10, 2013, New Orleans, LA. Abstract No. 3448
Background "Porter J, et al. Interim Results From a Phase 2A, Open-Label, Dose-Finding Study To Determine The Safety, Efficacy, And Tolerability of Sotatercept (ACE-001) In Adults with Beta-Thalassemia. Presented at the 19thCongress of the European Hematology Association, June 12-15, 2015, Milan, Italy. Abstract No. S622 "

Evaluation	Drug	Modality	Dose	Route
Subject	Sotatercept	Protein / enzyme biologic	0.1 mg/kg	Subcutaneous
Subject	Sotatercept	Protein / enzyme biologic	0.3 mg/kg	Subcutaneous
Subject	Sotatercept	Protein / enzyme biologic	0.5 mg/kg	Subcutaneous
Subject	Sotatercept	Protein / enzyme biologic	0.75 mg/kg	Subcutaneous
Subject	Sotatercept	Protein / enzyme biologic	1 mg/kg	Subcutaneous
Subject	Sotatercept	Protein / enzyme biologic	1.5 mg/kg	Subcutaneous