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

NCT ID: NCT03805789

Last Updated: 2025-11-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

222 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-27

Study Completion Date

2026-03-31

Brief Summary

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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).

Detailed Description

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Conditions

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Acute-graft-versus-host Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

PREVENTION

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AAT (low dose)

Open label. AAT is a lyophilized product for intravenous (IV) administration

Group Type EXPERIMENTAL

AAT

Intervention Type BIOLOGICAL

AAT is a lyophilized product for IV administration.

AAT (medium dose)

Open label. AAT is a lyophilized product for IV administration

Group Type EXPERIMENTAL

AAT

Intervention Type BIOLOGICAL

AAT is a lyophilized product for IV administration.

AAT (high dose)

Open label. AAT is a lyophilized product for IV administration

Group Type EXPERIMENTAL

AAT

Intervention Type BIOLOGICAL

AAT is a lyophilized product for IV administration.

AAT (selected dose from open-label)

Double-blind. AAT is a lyophilized product for IV administration

Group Type EXPERIMENTAL

AAT

Intervention Type BIOLOGICAL

AAT is a lyophilized product for IV administration.

Placebo

Albumin solution administered intravenously

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type BIOLOGICAL

Albumin solution administered intravenously

Interventions

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AAT

AAT is a lyophilized product for IV administration.

Intervention Type BIOLOGICAL

Placebo

Albumin solution administered intravenously

Intervention Type BIOLOGICAL

Other Intervention Names

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Alpha-1 proteinase inhibitor Respreeza

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Physician

Role: STUDY_DIRECTOR

CSL Behring

Locations

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Seoul National University Hospital

Seoul, , South Korea

Site Status

Hospital Universitario Valle de Hebron

Barcelona, , Spain

Site Status

HonorHealth Scottsdale Shea Medical Center

Scottsdale, Arizona, United States

Site Status

Johns Hopkins Hospital

St. Petersburg, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

University of Kansas Cancer Center

Westwood, Kansas, United States

Site Status

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

University of Michigan Medical Center

Ann Arbor, Michigan, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

University Hospital Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

The University of Texas-MD Anderson Cancer Center

San Antonio, Texas, United States

Site Status

University of Utah Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Site Status

Royal Brisbane and Women's Hospital

Herston, Queenland, Australia

Site Status

Uniklinik Köln

Cologne, , Germany

Site Status

University Hospital Catania

Calabria, Catania, Italy

Site Status

Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli

Calabria, , Italy

Site Status

Anjo Kosei Hospital

Anjo-shi, , Japan

Site Status

Tokyo Metropolitan Komagome Hospital

Bunkyō City, , Japan

Site Status

Hiroshima University Kasumi Campus

Hiroshima, , Japan

Site Status

Aichi Medical Center Nagoya Daiichi Hospital

Nagoya, , Japan

Site Status

Nagoya University Hospital

Nagoya, , Japan

Site Status

Okayama University Hospital

Okayama, , Japan

Site Status

Osaka International Cancer Institute

Osaka, , Japan

Site Status

Osaka Metropolitan University Hospital

Osaka, , Japan

Site Status

Hokkaido University Hospital

Sapporo, , Japan

Site Status

INJE University Haeundae Paik Hospital

Busan, , South Korea

Site Status

Pusan National University Hospital

Busan, , South Korea

Site Status

Gachon University Gil Medical Center

Incheon, , South Korea

Site Status

Marqués de Valdecilla University Hospital

Barcelona, , Spain

Site Status

Salamanca University Hospital

Salamanca, , Spain

Site Status

Ankara Abdurrahman Yurtaslan

Ankara, , Turkey (Türkiye)

Site Status

Turgut Ozal Medicine Center

Battalgazi, , Turkey (Türkiye)

Site Status

Countries

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Turkey (Türkiye) United States Australia Germany Italy Japan South Korea Spain

Other Identifiers

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2018-000329-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2024-511164-92-00

Identifier Type: CTIS

Identifier Source: secondary_id

CSL964_2001

Identifier Type: -

Identifier Source: org_study_id

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