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, multicenter, placebo-controlled study for prevention of acute GVHD (aGVHD) in participants undergoing an unrelated (matched or single allele mismatched) or matched related allogeneic hematopoietic cell transplantation (HCT).
Condition
- Acute-graft-versus-host Disease
Eligibility
- Eligible Ages
- Over 12 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Male or female participants, >=12 years of age (>= 18 years of age for participants at German sites only), undergoing HCT for hematological malignancies, including leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms. - Planned myeloablative conditioning regimen. - Participants must have a related or unrelated donor as follows: - Related donor must be a 6 / 6 match for human leukocyte antigen (HLA)-A, -B, at intermediate (or higher) resolution, and -DR beta 1 (DRB1) at high resolution using deoxyribonucleic acid (DNA)-based typing. - Unrelated donor must be 7 / 8 or 8 / 8 match for HLA-A, -B, and -C at intermediate (or higher) resolution, and -DRB1 at high resolution using DNA-based typing.
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 transplant. - Planned use of cyclophosphamide after HCT for GVHD prophylaxis. - Planned haploidentical donor.
Study Design
- Phase
- Phase 2/Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Prevention
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental AAT (low dose) |
Open label. AAT is a lyophilized product for intravenous (IV) administration |
|
|
Experimental AAT (medium dose) |
Open label. AAT is a lyophilized product for IV administration |
|
|
Experimental AAT (high dose) |
Open label. AAT is a lyophilized product for IV administration |
|
|
Experimental AAT (selected dose from open-label) |
Double-blind. AAT is a lyophilized product for IV administration |
|
|
Placebo Comparator Placebo |
Albumin solution administered intravenously |
|
Recruiting Locations
University of Kansas Cancer Center
Westwood 4281639, Kansas 4273857 66205
Westwood 4281639, Kansas 4273857 66205
Contact:
Use Central Contact
Use Central Contact
More Details
- Status
- Recruiting
- Sponsor
- CSL Behring