NCT04167514

Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 136 (actual)
• Sponsor: CSL Behring · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Graft Versus Host Disease (GVHD)

Interventions

Type	Name	Description
BIOLOGICAL	Alpha-1 antitrypsin (AAT)	AAT is a lyophilized product for intravenous administration
DRUG	Placebo	Albumin solution administered intravenously

Timeline

• Start date: 2020-01-09
• Primary completion: 2023-09-11
• Completion: 2024-06-26
• First posted: 2019-11-19
• Last updated: 2025-08-29
• Results posted: 2025-08-29

Locations

25 sites across 1 country: United States

Regulatory

• FDA-regulated drug study