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: March 14, 2025
Sponsor: CSL Behring
Overall Status: Active - 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 for participantsat German sites only), undergoing HCT for hematological malignancies, includingleukemia, 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 resolutionusing deoxyribonucleic acid (DNA)-based typing.

  • Unrelated donor must be 7 / 8 or 8 / 8 match for HLA-A, -B, and -C atintermediate (or higher) resolution, and -DRB1 at high resolution usingDNA-based typing.

Exclusion

Exclusion Criteria:

  • Prior autologous or allogeneic HCT.

  • T cell depleted transplant or planned use of anti-T cell antibody therapy either exvivo 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: 310
Treatment Group(s): 3
Primary Treatment: Placebo
Phase: 2/3
Study Start date:
March 27, 2019
Estimated Completion Date:
October 18, 2029

Connect with a study center

  • Royal Brisbane and Women's Hospital

    Herston, Queenland 4029
    Australia

    Active - Recruiting

  • University Essen

    Essen, 45147
    Germany

    Site Not Available

  • Uniklinik Köln, lnnere Mediz

    Köln, 50937
    Germany

    Active - Recruiting

  • University Hospital Catania

    Calabria, Catania 95123
    Italy

    Active - Recruiting

  • Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli

    Calabria, 89133
    Italy

    Active - Recruiting

  • Anjo Kosei Hospital

    Anjo-shi, 4668602
    Japan

    Site Not Available

  • Tokyo Metropolitan Komagome Hospital

    Bunkyo-ku, 1138677
    Japan

    Site Not Available

  • Hiroshima University Kasumi Campus

    Hiroshima, 7348551
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya, 4668550
    Japan

    Site Not Available

  • Aichi Medical Center Nagoya Daiichi Hospital

    Nagoya-shi, 4538511
    Japan

    Site Not Available

  • Okayama University Hospital

    Okayama-shi, 71008558
    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

  • INJE University Haeundae Paik Hospital

    Busan, 48108
    Korea, Republic of

    Active - Recruiting

  • Pusan National University Hospital

    Busan, 49241
    Korea, Republic of

    Active - Recruiting

  • Gachon University Gil Medical Center

    Incheon, 21565
    Korea, Republic of

    Active - Recruiting

  • Seoul National University Hospital

    Seoul, 03080
    Korea, Republic of

    Active - Recruiting

  • 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

    Active - Recruiting

  • Hospital Regional Universitario de Malaga

    Málaga, 29010
    Spain

    Site Not Available

  • Salamanca University Hospital

    Salamanca, 37007
    Spain

    Active - Recruiting

  • Universitario de Salamanca

    Salamanca, 37007
    Spain

    Active - Recruiting

  • Hospital Universitario Marqués

    Santander,
    Spain

    Site Not Available

  • Ankara Abdurrahman Yurtaslan

    Ankara, 06200
    Turkey

    Active - Recruiting

  • Turgut Ozal Medicine Center

    Battalgazi, 44280
    Turkey

    Active - Recruiting

  • The Christie NHS Foundation

    Manchester, M20 4BX
    United Kingdom

    Site Not Available

  • HonorHealth Scottsdale Shea Medical Center

    Scottsdale, Arizona 85258
    United States

    Active - Recruiting

  • Scottsdale HH Cancer Transplant

    Scottsdale, Arizona 85258
    United States

    Active - Recruiting

  • Johns Hopkins Hospital

    Saint Petersburg, Florida 33701
    United States

    Active - Recruiting

  • Emory University

    Atlanta, Georgia 30322
    United States

    Active - Recruiting

  • University of Kansas Cancer Center

    Westwood, Kansas 66205
    United States

    Active - Recruiting

  • Johns Hopkins Hospital

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Dana Farber Cancer Institute

    Boston, Massachusetts 02215
    United States

    Active - Recruiting

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • University of Michigan Medical Center

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • Mount Sinai Medical Center

    New York, New York 10029
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27705
    United States

    Active - Recruiting

  • 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

    Active - Recruiting

  • 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

    Active - Recruiting

  • 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 Virginia Health System

    Charlottesville, Virginia 22903
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Site Not Available

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington 98109
    United States

    Active - Recruiting

  • Seattle Cancer Care Alliance

    Seattle, Washington 98109
    United States

    Active - Recruiting

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