NCT01523821

Alpha 1 Anti-Trypsin (AAT) in Treating Patients With Acute Graft-Versus-Host Disease GVHD)

Treatment of Steroid Non-responsive Acute GVHD With Alpha 1 Antitrypsin (AAT). A Phase I/II Study

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase I/II trial evaluates the efficacy and adverse effects of alpha 1 anti-trypsin (AAT) for the treatment of acute graft-versus-host disease (GVHD) after hematopoietic stem cell transplantation.

Detailed description

PRIMARY OBJECTIVES: I. Determine the safety and tolerability of AAT in patients with steroid non-responsive acute GVHD. II. Characterize pharmacodynamic effects of AAT on pro-inflammatory cytokines, heparan sulfate, and the spectrum of peripheral blood T cells. III. Determine clinical responses of GVHD to AAT in patients with steroid non-responsive acute GVHD. OUTLINE: This is a phase I/II dose-escalation study of AAT. Patients will receive AAT intravenously (IV) on study days 1, 3, 5, and 7. Patients who experience no toxicity and in whom GVHD is stable or improved after the day 7 dose can continue therapy with AAT on days 9, 11, 13 and 15 for a total of 8 doses.

Conditions

• Graft-Versus-Host Disease (GVHD) Acute on Chronic

Interventions

Type	Name	Description
DRUG	Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]	Alpha 1-Proteinase Inhibitor, Human 1 MG \[Glassia\] at various levels over different days

Timeline

• Start date: 2013-10-11
• Primary completion: 2017-01-15
• Completion: 2017-01-15
• First posted: 2012-02-01
• Last updated: 2018-10-30
• Results posted: 2018-10-30

Locations

1 site across 1 country: United States