Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone

NCT ID: NCT04167514

Last Updated: 2025-08-29

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

136 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-09

Study Completion Date

2024-06-26

Brief Summary

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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

Detailed Description

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Conditions

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Graft Versus Host Disease (GVHD)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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AAT

Alpha-1 antitrypsin (AAT) is a lyophilized powder for intravenous administration

Group Type EXPERIMENTAL

Alpha-1 antitrypsin (AAT)

Intervention Type BIOLOGICAL

AAT is a lyophilized product for intravenous administration

Placebo

Albumin solution administered intravenously

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Albumin solution administered intravenously

Interventions

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Alpha-1 antitrypsin (AAT)

AAT is a lyophilized product for intravenous administration

Intervention Type BIOLOGICAL

Placebo

Albumin solution administered intravenously

Intervention Type DRUG

Other Intervention Names

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Alpha-1 proteinase inhibitor (A1-P1)

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Blood and Marrow Transplant Clinical Trials Network

NETWORK

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

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Stanford University

Stanford, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Moffitt Cancer Center

Tampa, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Northside Hospital

Atlanta, Georgia, United States

Site Status

IU Hospital

Indianapolis, Indiana, United States

Site Status

Univ. of Kansas Cancer Center

Westwood, Kansas, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

U-M Medical Center

Ann Arbor, Michigan, United States

Site Status

Karmanos Cancer Center

Detroit, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

MSKCC

New York, New York, United States

Site Status

Levine Cancer Center

Charlotte, North Carolina, United States

Site Status

Duke

Durham, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Ohio State Univ.Medical Center

Columbus, Ohio, United States

Site Status

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States

Site Status

Center for Gene Therapy

Houston, Texas, United States

Site Status

MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Virginia Commonwealth Univ.

Richmond, Virginia, United States

Site Status

Fred Hutchinson Clinic

Seattle, Washington, United States

Site Status

University of Wisconsin

Madison, Wisconsin, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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BMT CTN Protocol 1705

Identifier Type: OTHER

Identifier Source: secondary_id

CSL964_5001 / BMT CTN 1705

Identifier Type: -

Identifier Source: org_study_id

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