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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
QUADRUPLE
Enrollment
222
The time to Grade II-IV aGVHD or death
Acute GVHD will be assessed using the Harris scoring system.
Time frame: Through 180 days after HCT
Proportion of participants with lower gastrointestinal (GI) aGVHD or Grade III-IV aGVHD in any organ
Time frame: Through 180 days after HCT
Proportion of participants with severe infections defined by National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) greater than or equal to (>=) Grade 3
Time frame: Through Day 60 after HCT
Proportion of participants with Grade II-IV aGVHD or death
Time frame: Through 100 days and 180 days after HCT
Proportion of participants with lower GI aGVHD
Time frame: Through Days 60, 100 and 180 after HCT
Proportion of participants with severe infections defined by NCI-CTCAE >= Grade 3
Time frame: Through 100 and 180 days after HCT
Number of deaths (relapse and nonrelapse-related)
Death by any cause
Time frame: Within 180, 365, and 730 days after HCT
Proportion of participants with Grade III-IV aGVHD or death
Time frame: Through Days 60, 100, and 180 days after HCT
Proportion of participants with moderate to severe chronic GVHD
Moderate to severe chronic GVHD graded according to National Institutes of Health (NIH) scale.
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HonorHealth Scottsdale Shea Medical Center
Scottsdale, Arizona, United States
Johns Hopkins Hospital
St. Petersburg, Florida, United States
Emory University
Atlanta, Georgia, United States
University of Kansas Cancer Center
Westwood, Kansas, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
University of Michigan Medical Center
Ann Arbor, Michigan, United States
Duke University Medical Center
Durham, North Carolina, United States
University Hospital Cleveland Medical Center
Cleveland, Ohio, United States
The University of Texas-MD Anderson Cancer Center
San Antonio, Texas, United States
University of Utah Primary Children's Hospital
Salt Lake City, Utah, United States
...and 24 more locations
Time frame: Within 180, 365, 545, and 730 days after HCT
Proportion of participants who have discontinued immune suppression therapies including standard of care GVHD prophylaxis and steroid treatment
Time frame: Within 180 and 365 days after HCT
Time to neutrophil engraftment
Time to the first of 3 consecutive days of absolute neutrophil counts ≥ 500/µL.
Time frame: Through 365 days after HCT
Time to GVHD relapse-free survival
GVHD free, relapse free, survival defined as time to any of the following events: 1) Grade III-IV acute GVHD, 2) moderate-severe chronic GVHD, 3) primary malignancy relapse or 4) death.
Time frame: Within 365 and 730 days after HCT
Proportion of participants with relapse of primary malignancies
Time frame: Through 180, 365, and 730 days after HCT
Proportion of participants with Grade II-IV aGVHD with an overall (complete + partial) response, complete response and partial response
Time frame: Approximately 4 weeks after the initiation of systemic steroids during 8-week Treatment Period
Percent of participants with study drug related adverse events
Time frame: Up to 365 days after HCT
Maximum concentration (Cmax) of AAT
Time frame: Before and up to 72 after infusion of AAT
Area under the concentration curve (AUC) for AAT
Time frame: Before and up to 72 after infusion of AAT
Ctrough of AAT
Time frame: Before and up to 72 after infusion of AAT
Clearance (CL) of AAT
Time frame: Before and up to 72 after infusion of AAT
Volume of distribution (V) for AAT
Time frame: Before and up to 72 after infusion of AAT