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A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Trial of Alpha 1 Antitrypsin (AAT) Combined with Corticosteroids vs Corticosteroids Alone for the Treatment of High Risk Acute Graft-versus-Host Disease (GVHD) Following Allogeneic Hematopoietic Stem Cell Transplant
Study CSL964_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD
The primary objective of this trial is to compare the rate of complete response (CR) and partial response (PR) on Day 28 post-randomization between AAT and CS versus placebo to match (PTM) and CS in patients with high-risk acute GVHD. Secondary objectives are to assess the following: 1. Duration of response at 6 and 12 months postrandomization. 2. Cumulative incidence of non-relapse mortality (NRM) at 6 and 12 months post-randomization. 3. Overall survival (OS) and progression free survival (PFS) at 6 and 12 months post-randomization. 4. GVHD-free survival at Day 56 post-randomization. 5. Proportions of CR, very good partial response (VGPR), PR, and treatment failure (TF) at Day 7, 14, 21, 28, 56, and 86 post-randomization. 6. Incidence of systemic infections to assess safety. 7. Incidence of Adverse Events (AEs) at 30 days post last dose of drug to assess safety. 8. Incidence of chronic GVHD at 6 and 12 months post randomization and stem cell infusion (Day 0 HCT). 9. Incidence of disease relapse at 6 and 12 months post randomization.
Alpha 1 - Antitrypsin (); Methylprednisolone (); Placebo (); prednisone ()