Clinical trial NCT04167514: Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone (BMT CTN 1705)
Official title: A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Trial of Alpha 1 - Antitrypsin (AAT) Combined With Corticosteroids vs Corticosteroids Alone for the Treatment of High Risk Acute Graft-versus-Host Disease (GVHD) Following Allogeneic Hematopoietic Stem Cell Transplant (BMT CTN 1705)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Graft Versus Host Disease (GVHD)
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
Collaborators: name: Blood and Marrow Transplant Clinical Trials Network
name: National Institutes of Health (NIH)
name: National Heart, Lung, and Blood Institute (NHLBI)
name: National Cancer Institute (NCI)
First posted: 2019-11-19
Enrollment: 136
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Study CSL964_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD


Eligibility criteria: Inclusion Criteria:

* Patients 12 years of age or older
* Initial presentation of acute GVHD after allogeneic hematopoietic cell transplantation for any indication
* Any graft or donor source or conditioning intensity
* Clinical diagnosis of acute GVHD requiring systemic therapy with corticosteroids

Exclusion Criteria:

* Prior exogenous AAT exposure for GVHD prophylaxis
* Relapsed, progressing, or persistent malignancy
* de novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment
* Receiving other drugs for the treatment of GVHD
* Receiving systemic CS for any indication within 7 days before the onset of acute GVHD

Primary outcomes:
measure: Percent of participants with complete or partial response to acute Graft-versus-Host Disease (GVHD) treatment
timeFrame: 28 days post-randomization
Secondary outcomes:
measure: Duration of response (DOR)
timeFrame: up to 12 months post-randomization
measure: Percent of participants with non-relapse mortality (NRM)
timeFrame: up to 12 months post-randomization
measure: Percent of participants with overall survival and progression-free survival
timeFrame: up to 12 months post-randomization
measure: Percent of participants with GVHD-free survival
timeFrame: Day 56 post-randomization
measure: Percent of participants with response
timeFrame: At Day 7, 14, 21, 28, 56, and 86 post-randomization
measure: Percent of participants with Grade 2 to 3 systemic infections
timeFrame: up to 30 days after the last dose of study drug
measure: Percent of participants with Grade 3 to 5 treatment-emergent adverse events (TEAEs)
timeFrame: up to 30 days after the last dose of study drug
measure: Percent of participants with chronic GVHD
timeFrame: up to 12 months post-randomization
measure: Percent of participants with disease relapse
timeFrame: up to 12 months post-randomization
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Tampa
country: United States
facility: Moffitt Cancer Center
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
city: Indianapolis
country: United States
facility: IU Hospital
state: Indiana
city: Westwood
country: United States
facility: Univ. of Kansas Cancer Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: U-M Medical Center
state: Michigan
city: Detroit
country: United States
facility: Karmanos Cancer Center
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: New York
country: United States
facility: MSKCC
state: New York
city: Charlotte
country: United States
facility: Levine Cancer Center
state: North Carolina
city: Durham
country: United States
facility: Duke
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: Ohio State Univ.Medical Center
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt-Ingram Cancer Center
state: Tennessee
city: Houston
country: United States
facility: Center for Gene Therapy
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth Univ.
state: Virginia
city: Seattle
country: United States
facility: Fred Hutchinson Clinic
state: Washington
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: