Graft-Versus-Host Disease (GVHD) Acute on Chronic Clinical Trial
Official title:
Treatment of Steroid Non-responsive Acute GVHD With Alpha 1 Antitrypsin (AAT). A Phase I/II Study
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.
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.
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