Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)

Last updated: December 7, 2022
Sponsor: Medical College of Wisconsin
Overall Status: Completed

Phase

3

Condition

Leukemia

Red Blood Cell Disorders

White Cell Disorders

Treatment

N/A

Clinical Study ID

NCT00075816
BMTCTN0201
5U24CA076518
BMT CTN 0201
U01HL069294-05
  • Ages < 66
  • All Genders

Study Summary

The study is designed as a Phase III, randomized, open label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells (PBSC) versus marrow from unrelated donors for transplantation in patients with hematologic malignancies. Recipients will be stratified by transplant center and disease risk and will be randomized to either the PBSC or marrow arm in a 1:1 ratio.

Eligibility Criteria

Inclusion

Patient Inclusion Criteria: One of the following diagnoses:

  • Acute myelogenous leukemia at the following stages: first remission, second remission,third or subsequent remission, or not in remission
  • Acute lymphoblastic leukemia at the following stages: first remission, secondremission, third or subsequent remission, or not in remission
  • Chronic myelogenous leukemia at the following stages: chronic phase, acceleratedphase, or blast phase
  • Myelodysplastic syndromes (MDS) at the following stages: refractory anemia; refractoryanemia with ringed sideroblasts; refractory cytopenia with multilineage dysplasia;refractory cytopenia with multilineage dysplasia and ringed sideroblasts; refractoryanemia with excess blasts-1 (5-10% blasts); refractory anemia with excess blasts-2 (10-20% blasts); myelodysplastic syndrome, unclassified; or MDS associated withisolated del (5q)
  • Myeloproliferative diseases: chronic myelomonocytic leukemia; agnogenic myeloidmetaplasia with myelofibrosis (idiopathic myelofibrosis); juvenile myelomonocyticleukemia
  • Therapy-related acute myelogenous leukemia (AML) or MDS with prior malignancy that hasbeen in remission for at least 12 months. If the remission is less than 12 months,Medical Monitor or Protocol Chair approval is required for eligibility

Exclusion

Patient Exclusion Criteria:

  • Prior allogeneic or autologous transplants using any hematopoietic stem cell source;patients with secondary malignancies who have had a prior autologous transplant willbe eligible; the prior autologous transplant must have been performed for the primarymalignancy (such as lymphoma) and must have occurred 12 or more months prior toenrollment
  • Lymphoma (11% of 2001 NMDP transplants), other malignant disorders (6%), andnon-malignant disorders (9%) Donor Inclusion Criteria:
  • Matched for HLA-A, B, and DRB1 antigens
  1. One antigen mismatch at HLA-A, B, or DRB1 is acceptable with or without mismatchat HLA-C
  2. Typing is by DNA techniques: intermediate resolution for A, B, and C, and highresolution for DRB1. HLA-C typing is mandatory but will not count in the match.
  • Willing to undergo both bone marrow harvest and G-CSF administration with apheresis
  • Willing to be randomly assigned to either marrow or PBSC collection
  • Adequate peripheral venous access for leukapheresis or willing to undergo placement ofa central catheter
  • Donor center affiliation with NMDP
  • Additional donor inclusion criteria can be found in the Donor Companion Manual Donor Exclusion Criteria:
  • Pregnant (positive serum β-HCG) or uninterruptible breastfeeding
  • Known allergy to G-CSF or to E. Coli-derived recombinant protein products
  • History of autoimmune disorders
  • History of deep vein thrombosis or venous thromboembolism
  • History of iritis or episcleritis
  • History of serious adverse reaction to anesthesia
  • Thrombocytopenia (platelets less than 150,000 per mcL) at baseline evaluation
  • Current treatment with lithium
  • Presence of sickle hemoglobin as demonstrated by appropriate testing such ashemoglobin electrophoresis
  • Receiving experimental therapy or investigational agents

Study Design

Total Participants: 551
Study Start date:
January 01, 2004
Estimated Completion Date:
April 30, 2014

Study Description

BACKGROUND:

Many studies of allogeneic marrow transplantation have shown that a higher dose of marrow cells correlates with more robust hematopoietic engraftment and lower mortality from infectious complications. Peripheral blood stem cells (PBSC) collected after mobilization with granulocyte colony stimulating factor (G-CSF) contain a larger number of CD34-positive (CD34) progenitors and total cells than bone marrow. These observations led to the hypothesis that transplantation of PBSC would lead to lower mortality compared to transplantation of marrow. In addition, PBSC grafts have a higher T cell content, predicting a possibly more powerful anti-leukemia effect. However, the higher T cell content of PBSC may also lead to increased incidence and severity of acute and chronic graft-versus-host disease (GVHD). This concern is especially serious when the donor is unrelated to the recipient. This prospective, randomized, multicenter clinical trial of unrelated donor transplantation will test the hypothesis that transplantation of PBSC leads to similar patient survival compared to transplantation of marrow.

DESIGN NARRATIVE:

This is a Phase III randomized, open label, multicenter clinical trial sponsored by the National Marrow Donor Program (NMDP) and the National Institutes of Health (NIH). The objective of the trial is to test the null hypothesis that there is no difference in overall survival after PBSC versus marrow transplants from HLA compatible unrelated donors. The study will compare G-CSF-mobilized PBSC transplantation with bone marrow transplantation from HLA-compatible unrelated donors for patients with leukemia, myelodysplastic or myeloproliferative syndromes. Conditioning and GVHD prophylaxis regimens will vary by center and within centers, however, the center must declare before randomization what regimens will be used for each patient. The primary endpoint of this trial is 2-year survival following randomization. Secondary analyses will consider neutrophil and platelet recovery, acute and chronic GVHD, time off all immunosuppressive therapy, relapse, infections, adverse events and immune reconstitution. The trial will include evaluation of patient and donor quality of life, composition of the graft, and immune reconstitution. Accrual is anticipated for 3 years with a follow-up period of 3 years.

Connect with a study center

  • Tom Baker Cancer Centre, Calgary

    Calgary, Alberta T2N 4N2
    Canada

    Site Not Available

  • Vancouver General Hospital

    Vancouver, British Columbia V5Z1M9
    Canada

    Site Not Available

  • Hamilton Health Sciences - McMaster Site

    Hamilton, Ontario
    Canada

    Site Not Available

  • Ottawa Hospital

    Ottawa, Ontario
    Canada

    Site Not Available

  • University of Toronto, Princess Margaret Hospital

    Toronto, Ontario M5G 2M9
    Canada

    Site Not Available

  • Queen Elizabeth II Health Sciences Centre - Halifax

    Halifax,
    Canada

    Site Not Available

  • University of Alabama at Birmingham

    Birmingham, Alabama 35294
    United States

    Site Not Available

  • City of Hope National Medical Center

    Duarte, California 91010
    United States

    Site Not Available

  • UCSD Cancer Center

    La Jolla, California 92093-0960
    United States

    Site Not Available

  • University of California, San Francisco

    San Francisco, California 94143
    United States

    Site Not Available

  • Stanford Hospital and Clinics

    Stanford, California 94305
    United States

    Site Not Available

  • University of Florida College of Medicine (Shands)

    Gainesville, Florida 32610-100277
    United States

    Site Not Available

  • Emory University

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Loyola University

    Maywood, Illinois 60153
    United States

    Site Not Available

  • Indiana BMT at Beech Grove

    Beech Grove, Indiana 46107
    United States

    Site Not Available

  • IBMT (Indiana Blood and Marrow Transplant) at St Francis Franciscan Health

    Indianapolis, Indiana 46237
    United States

    Site Not Available

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • University of Kansas Hospital

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • University of Maryland

    Baltimore, Maryland 21228
    United States

    Site Not Available

  • DFCI/Brigham & Women's

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • University of Michigan Medical Center

    Ann Arbor, Michigan 48109-0942
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • Mayo Clinic Cancer Center

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • Washington University/Barnes Jewish Hospital

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Washington University/St. Louis Children's Hospital

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • University of Nebraska Medical Center

    Omaha, Nebraska 68198-3330
    United States

    Site Not Available

  • Hackensack University Medical Center Cancer Center

    Hackensack, New Jersey 07601
    United States

    Site Not Available

  • Roswell Park Cancer Institute

    Buffalo, New York 14263-0001
    United States

    Site Not Available

  • Cohen Children's Hospital

    New Hyde Park, New York 11040
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina 27157-1082
    United States

    Site Not Available

  • Ohio State/Arthur G. James Cancer Hospital

    Columbus, Ohio 43210
    United States

    Site Not Available

  • University of Oklahoma Medical Center

    Oklahoma City, Oklahoma 73104
    United States

    Site Not Available

  • Oregon Health & Science University (Peds)

    Portland, Oregon 97239-3098
    United States

    Site Not Available

  • Oregon Health Sciences University

    Portland, Oregon 97239
    United States

    Site Not Available

  • University of Pennsylvania Cancer Center

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Pittsburgh Cancer Institute

    Pittsburgh, Pennsylvania 15232
    United States

    Site Not Available

  • Vanderbilt University

    Nashville, Tennessee 37232-6838
    United States

    Site Not Available

  • Baylor University Medical Center

    Dallas, Texas 77030
    United States

    Site Not Available

  • Baylor College of Medicine/The Methodist Hospital

    Houston, Texas 77030-2399
    United States

    Site Not Available

  • University of Texas/MD Anderson CRC

    Houston, Texas 77030-4009
    United States

    Site Not Available

  • Texas Transplant Institute

    San Antonio, Texas 78229
    United States

    Site Not Available

  • Utah BMT/Primary Children's Medical Center

    Salt Lake City, Utah 84112
    United States

    Site Not Available

  • Utah BMT/University of Utah Medical School

    Salt Lake City, Utah 84132
    United States

    Site Not Available

  • Virginia Commonwealth University MCV Hospitals

    Richmond, Virginia 23298-0037
    United States

    Site Not Available

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington 98104
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.