A Proof of Concept Pilot Trial of Alpha-1-Antitrypsin for Pre-Emption Of Steroid-Refractory Acute GVHD
Status:
Completed
Trial end date:
2020-08-21
Target enrollment:
Participant gender:
Summary
Bone marrow transplant (BMT) patients can develop graft-versus-host disease (GVHD), a serious
and potentially fatal complication. The researchers have developed a blood test to identify
patients most at risk for developing severe GVHD. Patients who consent to this study will
have their blood tested up to two times after BMT to determine if they are at high risk for
severe GVHD. The tests will be performed one week and two weeks after BMT. Patients who are
high risk will be treated with a drug called alpha-1-antitrypsin (AAT) to see if it prevents
the development of severe GVHD. Patients will receive 16 doses of AAT through a catheter
placed into a blood vessel over eight weeks. AAT will be given either in the hospital or the
outpatient clinic two times per week. Patients will be followed for the development of severe
GVHD for up to four months from the BMT and will continue to be followed at routine clinic
visits for up to one year after BMT.