This phase II trial studies the side effects and how well alpha-1-proteinase inhibitor human works in preventing development of graft versus host disease in stem cell transplant participants. Graft versus host disease is the most common serious complication after receiving a bone marrow transplant. Alpha-1-proteinase inhibitor human may help prevent the inflammatory immune response from graft versus host disease and damage to the skin, liver, stomach, and/or intestines.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT03459040.
PRIMARY OBJECTIVES
I. To generate data for assessing the feasibility, safety and preliminary efficacy of alpha-1-proteinase inhibitor human (alpha-1-antitrypsin [AAT]) as pre-emptive therapy in patients at high risk for the development of steroid-refractory graft versus host disease (GVHD).
SECONDARY OBJECTIVES:
I. To generate a preliminary estimate of the 100 days incidence of clinically relevant GVHD states including steroid-refractory GVHD, grade II-IV GVHD, and grade III-IV in patients at high risk for the development of steroid-refractory GVHD treated with AAT.
II. To generate a preliminary estimate of the non-relapse mortality, relapse, and survival rates in patients at high risk for the development of steroid-refractory GVHD treated with AAT.
III. To generate a preliminary estimate of the incidence of severe toxicities, serious infections, and viral reactivations in patients at high risk for the development of steroid-refractory GVHD treated with AAT.
OUTLINE:
Participants receive alpha-1-proteinase inhibitor human intravenously (IV) over 11-22 minutes beginning on day 0 twice weekly for up to 16 doses over 8 weeks in the absence of disease progression or unacceptable toxicity.
After completion of study, participants are followed up to 1 year.
Lead OrganizationIcahn School of Medicine at Mount Sinai
Principal InvestigatorJohn Eric Levine
