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

Last updated: November 3, 2025
Sponsor: CSL Behring
Overall Status: Active - Not Recruiting

Phase

2/3

Condition

N/A

Treatment

Placebo

Alpha-1 antitrypsin (AAT)

AAT

Clinical Study ID

NCT03805789
CSL964_2001
2018-000329-29
2024-511164-92-00
  • Ages > 12
  • All Genders

Study Summary

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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • • Male or female participants, >=12 years of age (>= 18 years of age forparticipants at German sites only), undergoing HCT for hematological malignancies,including leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome, andmyeloproliferative 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, atintermediate (or higher) resolution, and -DR beta 1 (DRB1) at high resolution usingdeoxyribonucleic 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

Exclusion Criteria:

  • • Prior autologous or allogeneic HCT.

  • • T cell depleted transplant or planned use of anti-T cell antibody therapy eitherex vivo or in vivo (ie, anti thymocyte globulin [ATG], alemtuzumab) for GVHDprophylaxis.

  • • Planned umbilical cord blood transplant.

  • • Planned use of cyclophosphamide after HCT for GVHD prophylaxis.

  • • Planned haploidentical donor.

Study Design

Total Participants: 222
Treatment Group(s): 3
Primary Treatment: Placebo
Phase: 2/3
Study Start date:
March 27, 2019
Estimated Completion Date:
March 31, 2026

Connect with a study center

  • Royal Brisbane and Women's Hospital

    Herston, Queenland 4029
    Australia

    Site Not Available

  • Royal Brisbane and Women's Hospital

    Herston 6931014, Queenland 4029
    Australia

    Site Not Available

  • Uniklinik Köln

    Cologne 2886242, 50937
    Germany

    Site Not Available

  • Uniklinik Köln, lnnere Mediz

    Cologne 2886242, 50937
    Germany

    Active - Recruiting

  • University Essen

    Essen, 45147
    Germany

    Site Not Available

  • Uniklinik Köln, lnnere Mediz

    Köln, 50937
    Germany

    Site Not Available

  • University Hospital Catania

    Calabria, Catania 95123
    Italy

    Active - Recruiting

  • University Hospital Catania

    Calabria 8969017, Catania 95123
    Italy

    Site Not Available

  • Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli

    Calabria, 89133
    Italy

    Site Not Available

  • Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli

    Calabria 8969017, 89133
    Italy

    Site Not Available

  • Anjo Kosei Hospital

    Anjo-shi, 4668602
    Japan

    Site Not Available

  • Tokyo Metropolitan Komagome Hospital

    Bunkyo-ku, 1138677
    Japan

    Site Not Available

  • Tokyo Metropolitan Komagome Hospital

    Bunkyō City 11790632, 1138677
    Japan

    Site Not Available

  • Hiroshima University Kasumi Campus

    Hiroshima, 7348551
    Japan

    Site Not Available

  • Hiroshima University Kasumi Campus

    Hiroshima 1862415, 7348551
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya, 4668550
    Japan

    Site Not Available

  • Aichi Medical Center Nagoya Daiichi Hospital

    Nagoya 1856057, 4538511
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya 1856057, 4668550
    Japan

    Site Not Available

  • Aichi Medical Center Nagoya Daiichi Hospital

    Nagoya-shi, 4538511
    Japan

    Site Not Available

  • Okayama University Hospital

    Okayama 1854383, 71008558
    Japan

    Site Not Available

  • Okayama University Hospital

    Okayama-shi, 71008558
    Japan

    Site Not Available

  • Osaka International Cancer Institute

    Osaka 1853909, 5418567
    Japan

    Site Not Available

  • Osaka Metropolitan University Hospital

    Osaka 1853909, 5458586
    Japan

    Site Not Available

  • Osaka International Cancer Institute

    Osaka-shi, 5418567
    Japan

    Site Not Available

  • Osaka Metropolitan University Hospital

    Osaka-shi, 5458586
    Japan

    Site Not Available

  • Hokkaido University Hospital

    Sapporo, 0608648
    Japan

    Site Not Available

  • Hokkaido University Hospital

    Sapporo 2128295, 0608648
    Japan

    Site Not Available

  • INJE University Haeundae Paik Hospital

    Busan, 48108
    Korea, Republic of

    Active - Recruiting

  • Pusan National University Hospital

    Busan, 49241
    Korea, Republic of

    Site Not Available

  • Gachon University Gil Medical Center

    Incheon, 21565
    Korea, Republic of

    Site Not Available

  • Seoul National University Hospital

    Seoul, 03080
    Korea, Republic of

    Site Not Available

  • INJE University Haeundae Paik Hospital

    Busan 1838524, 48108
    South Korea

    Site Not Available

  • Pusan National University Hospital

    Busan 1838524, 49241
    South Korea

    Site Not Available

  • Gachon University Gil Medical Center

    Incheon 1843564, 21565
    South Korea

    Site Not Available

  • Seoul National University Hospital

    Seoul 1835848, 03080
    South Korea

    Site Not Available

  • Complejo Hospitalario de Navarra

    Pamplona, Navarra 31008
    Spain

    Site Not Available

  • Hospital Universitario Vall d'

    Barcelona, 08035
    Spain

    Active - Recruiting

  • Hospital Universitario Valle de Hebron

    Barcelona, 08035
    Spain

    Active - Recruiting

  • Marqués de Valdecilla University Hospital

    Barcelona, 39008
    Spain

    Site Not Available

  • Hospital Universitario Valle de Hebron

    Barcelona 3128760, 08035
    Spain

    Site Not Available

  • Marqués de Valdecilla University Hospital

    Barcelona 3128760, 39008
    Spain

    Site Not Available

  • Hospital Regional Universitario de Malaga

    Málaga, 29010
    Spain

    Site Not Available

  • Salamanca University Hospital

    Salamanca, 37007
    Spain

    Site Not Available

  • Universitario de Salamanca

    Salamanca, 37007
    Spain

    Active - Recruiting

  • Salamanca University Hospital

    Salamanca 3111108, 37007
    Spain

    Site Not Available

  • Hospital Universitario Marqués

    Santander,
    Spain

    Site Not Available

  • Ankara Abdurrahman Yurtaslan

    Ankara, 06200
    Turkey

    Site Not Available

  • Turgut Ozal Medicine Center

    Battalgazi, 44280
    Turkey

    Site Not Available

  • Ankara Abdurrahman Yurtaslan

    Ankara 323786, 06200
    Turkey (Türkiye)

    Site Not Available

  • Turgut Ozal Medicine Center

    Battalgazi 7457829, 44280
    Turkey (Türkiye)

    Site Not Available

  • The Christie NHS Foundation

    Manchester, M20 4BX
    United Kingdom

    Site Not Available

  • HonorHealth Scottsdale Shea Medical Center

    Scottsdale, Arizona 85258
    United States

    Site Not Available

  • Scottsdale HH Cancer Transplant

    Scottsdale, Arizona 85258
    United States

    Active - Recruiting

  • HonorHealth Scottsdale Shea Medical Center

    Scottsdale 5313457, Arizona 5551752 85258
    United States

    Site Not Available

  • Johns Hopkins Hospital

    Saint Petersburg, Florida 33701
    United States

    Site Not Available

  • Johns Hopkins Hospital

    St. Petersburg 4171563, Florida 4155751 33701
    United States

    Site Not Available

  • Emory University

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Emory University

    Atlanta 4180439, Georgia 4197000 30322
    United States

    Site Not Available

  • University of Kansas Cancer Center

    Westwood, Kansas 66205
    United States

    Site Not Available

  • University of Kansas Cancer Center

    Westwood 4281639, Kansas 4273857 66205
    United States

    Site Not Available

  • Johns Hopkins Hospital

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Dana Farber Cancer Institute

    Boston, Massachusetts 02215
    United States

    Site Not Available

  • Dana Farber Cancer Institute

    Boston 4930956, Massachusetts 6254926 02215
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • University of Michigan Medical Center

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • University of Michigan Medical Center

    Ann Arbor 4984247, Michigan 5001836 48109
    United States

    Site Not Available

  • Mount Sinai Medical Center

    New York, New York 10029
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Duke University Medical Center

    Durham 4464368, North Carolina 4482348 27705
    United States

    Site Not Available

  • Cleveland Cancer Center (University Hospitals UH - Seidman Cancer Center Ireland Cancer Center)

    Cleveland, Ohio 44106
    United States

    Active - Recruiting

  • University Hospital Cleveland Medical Center

    Cleveland, Ohio 44106
    United States

    Site Not Available

  • University Hospital Cleveland Medical Center

    Cleveland 5150529, Ohio 5165418 44106
    United States

    Site Not Available

  • Centennial Medical Center (TriStar BMT)

    Nashville, Tennessee 37203
    United States

    Site Not Available

  • Methodist Hospital

    San Antonio, Texas 78229
    United States

    Active - Recruiting

  • The University of Texas-MD Anderson Cancer Center

    San Antonio, Texas 77030
    United States

    Site Not Available

  • The University of Texas-MD Anderson Cancer Center

    San Antonio 4726206, Texas 4736286 77030
    United States

    Site Not Available

  • Primary Children's Hospital

    Salt Lake City, Utah 84113
    United States

    Site Not Available

  • University of Utah Primary Children's Hospital

    Salt Lake City, Utah 84113
    United States

    Site Not Available

  • University of Utah Primary Children's Hospital

    Salt Lake City 5780993, Utah 5549030 84113
    United States

    Site Not Available

  • University of Virginia Health System

    Charlottesville, Virginia 22903
    United States

    Site Not Available

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Site Not Available

  • University of Virginia Health System

    Charlottesville 4752031, Virginia 6254928 22903
    United States

    Site Not Available

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington 98109
    United States

    Site Not Available

  • Seattle Cancer Care Alliance

    Seattle, Washington 98109
    United States

    Active - Recruiting

  • Fred Hutchinson Cancer Research Center

    Seattle 5809844, Washington 5815135 98109
    United States

    Site Not Available

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