The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant

This study is a phase 2 / 3 prospective, double-blind, randomized, multicenter, placebo-controlled study for prevention of acute GVHD (aGVHD) in participants undergoing an unrelated (matched or single allele mismatched) or matched related allogeneic hematopoietic cell transplantation (HCT). This study consisted of two parts. In Part 1, the dose of Alpha1-Proteinase Inhibitor (AAT) to be used in Part 2 was identified based on safety and pharmacokinetic data. The selected dose was subsequently evaluated in Part 2, where the primary objective was to assess its efficacy in preventing aGVHD following HCT.