The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-Versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant

Purpose

This study is a Phase 2/3 prospective, double-blind, randomized, multi-center, placebo-controlled study for prevention of acute GVHD (aGVHD) in subjects undergoing an allogeneic hematopoietic cell transplant (HCT).

Condition

  • Acute-graft-versus-host Disease

Eligibility

Eligible Ages
Over 12 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Male or female subjects, ≥12 years of age (≥ 18 years of age for subjects at German sites only), undergoing HCT for hematological malignancies, including leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome and myeloproliferative neoplasms - Planned myeloablative conditioning regimen

Exclusion Criteria

  • Prior autologous or allogeneic HCT - T-cell depleted transplant or planned use of anti-T cell antibody therapy either ex vivo or in vivo (ie, anti-thymocyte globulin [ATG], alemtuzumab) for GVHD prophylaxis - Planned umbilical cord blood (UCB) transplant

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Prevention
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
AAT (low dose) 		Open label. Alpha-1 antitrypsin (AAT) is a lyophilized product for intravenous administration
  • Biological: Alpha-1 antitrypsin (AAT)
    Alpha-1 antitrypsin is a lyophilized product for intravenous administration.
    Other names:
    • Alpha-1 proteinase inhibitor
Experimental
AAT (medium dose) 		Open label. AAT is a lyophilized product for intravenous administration
  • Biological: Alpha-1 antitrypsin (AAT)
    Alpha-1 antitrypsin is a lyophilized product for intravenous administration.
    Other names:
    • Alpha-1 proteinase inhibitor
Experimental
AAT (high dose) 		Open label. AAT is a lyophilized product for intravenous administration
  • Biological: Alpha-1 antitrypsin (AAT)
    Alpha-1 antitrypsin is a lyophilized product for intravenous administration.
    Other names:
    • Alpha-1 proteinase inhibitor
Experimental
AAT (selected dose from open-label) 		Double-blind. AAT is a lyophilized product for intravenous administration
  • Biological: Alpha-1 antitrypsin (AAT)
    Alpha-1 antitrypsin is a lyophilized product for intravenous administration.
    Other names:
    • Alpha-1 proteinase inhibitor
Placebo Comparator
Placebo 		Albumin solution administered intravenously
  • Biological: Placebo
    Albumin solution administered intravenously

Recruiting Locations

University of Kansas Cancer Center
Westwood, Kansas 66205
Contact:
Use Central Contact

More Details

Status
Recruiting
Sponsor
CSL Behring

Study Contact

Trial Registration Coordinator
610-878-4000
clinicaltrials@cslbehring.com