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LeukemiaPhase 3NCT00075816

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

A Phase 3 study testing Allogeneic bone marrow transplantation and Peripheral blood stem cell transplantation in people with Leukemia and Myeloproliferative Disorders, run by Medical College of Wisconsin at 45 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages Up to 66 years
What is being tested?
Allogeneic bone marrow transplantation and Peripheral blood stem cell transplantation
Could I receive a placebo?
No, everyone receives an active treatment
How many people
551 participants
Where
45 sitesin 2 countries
Study dates
Jan 2004to Apr 2014
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 66 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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, second remission, third or subsequent remission, or not in remission
  • Chronic myelogenous leukemia at the following stages: chronic phase, accelerated phase, or blast phase
  • Myelodysplastic syndromes (MDS) at the following stages: refractory anemia; refractory anemia with ringed sideroblasts; refractory cytopenia with multilineage dysplasia; refractory cytopenia with multilineage dysplasia and ringed sideroblasts; refractory anemia with excess blasts-1 (5-10% blasts); refractory anemia with excess blasts-2 (10-20% blasts); myelodysplastic syndrome, unclassified; or MDS associated with isolated del (5q)
  • Myeloproliferative diseases: chronic myelomonocytic leukemia; agnogenic myeloid metaplasia with myelofibrosis (idiopathic myelofibrosis); juvenile myelomonocytic leukemia
  • Therapy-related acute myelogenous leukemia (AML) or MDS with prior malignancy that has been 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

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 will be eligible; the prior autologous transplant must have been performed for the primary malignancy (such as lymphoma) and must have occurred 12 or more months prior to enrollment
  • Lymphoma (11% of 2001 NMDP transplants), other malignant disorders (6%), and non-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 mismatch at HLA-C
  2. Typing is by DNA techniques: intermediate resolution for A, B, and C, and high resolution 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 of a 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 as hemoglobin electrophoresis
  • Receiving experimental therapy or investigational agents

Trial locations and contacts

45 sites in 2 countries.

45 sites in 2 countries

United States39 sites

  • University of Michigan Medical Center

    Ann Arbor, Michigan, 48109-0942

  • Emory University

    Atlanta, Georgia, 30322

  • University of Maryland

    Baltimore, Maryland, 21228

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • DFCI/Brigham & Women's

    Boston, Massachusetts, 02114

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263-0001

  • Ohio State/Arthur G. James Cancer Hospital

    Columbus, Ohio, 43210

  • Baylor University Medical Center

    Dallas, Texas, 77030

  • City of Hope National Medical Center

    Duarte, California, 91010

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • University of Florida College of Medicine (Shands)

    Gainesville, Florida, 32610-100277

  • Hackensack University Medical Center Cancer Center

    Hackensack, New Jersey, 07601

  • Baylor College of Medicine/The Methodist Hospital

    Houston, Texas, 77030-2399

  • University of Texas/MD Anderson CRC

    Houston, Texas, 77030-4009

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

    Indianapolis, Indiana, 46237

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • University of Kansas Hospital

    Kansas City, Kansas, 66160

  • UCSD Cancer Center

    La Jolla, California, 92093-0960

  • Loyola University

    Maywood, Illinois, 60153

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • Vanderbilt University

    Nashville, Tennessee, 37232-6838

  • Cohen Children's Hospital

    New Hyde Park, New York, 11040

  • University of Oklahoma Medical Center

    Oklahoma City, Oklahoma, 73104

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198-3330

  • University of Pennsylvania Cancer Center

    Philadelphia, Pennsylvania, 19104

  • University of Pittsburgh Cancer Institute

    Pittsburgh, Pennsylvania, 15232

  • Oregon Health & Science University (Peds)

    Portland, Oregon, 97239-3098

  • Oregon Health Sciences University

    Portland, Oregon, 97239

  • Virginia Commonwealth University MCV Hospitals

    Richmond, Virginia, 23298-0037

  • Mayo Clinic Cancer Center

    Rochester, Minnesota, 55905

  • Utah BMT/Primary Children's Medical Center

    Salt Lake City, Utah, 84112

  • Utah BMT/University of Utah Medical School

    Salt Lake City, Utah, 84132

  • Texas Transplant Institute

    San Antonio, Texas, 78229

  • University of California, San Francisco

    San Francisco, California, 94143

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98104

  • Washington University/Barnes Jewish Hospital

    St Louis, Missouri, 63110

  • Washington University/St. Louis Children's Hospital

    St Louis, Missouri, 63110

  • Stanford Hospital and Clinics

    Stanford, California, 94305

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157-1082

Other trials in United States

Canada6 sites

  • Tom Baker Cancer Centre, Calgary

    Calgary, Alberta, T2N 4N2

  • Queen Elizabeth II Health Sciences Centre - Halifax

    Halifax

  • Hamilton Health Sciences - McMaster Site

    Hamilton, Ontario

  • Ottawa Hospital

    Ottawa, Ontario

  • University of Toronto, Princess Margaret Hospital

    Toronto, Ontario, M5G 2M9

  • Vancouver General Hospital

    Vancouver, British Columbia, V5Z1M9

Other trials in Canada

About this study

Official title: A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From Human Leukocyte Antigen (HLA) Compatible Unrelated Donors (BMT CTN #0201)

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.

Read the detailed 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.

Being tested
Allogeneic bone marrow transplantation, Peripheral blood stem cell transplantation
Medical subject headings
Leukemia, Myeloproliferative Disorders, Myelodysplastic-Myeloproliferative Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Allogeneic bone marrow transplantationBiological

    Bone marrow transplant from HLA compatible unrelated donors.

  • Peripheral blood stem cell transplantationBiological

    Peripheral blood transplant from HLA compatible unrelated donors.

Study groups in NCT00075816
Study groupRoleWhat they receive
Bone Marrow TransplantActive comparatorAllogeneic bone marrow transplantation
Receives: Biological: Allogeneic bone marrow transplantation
Blood Stem Cell TransplantActive comparatorPeripheral blood stem cell transplantation
Receives: Biological: Peripheral blood stem cell transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Two-year Overall Survival

    Overall survival rate at 2 years according to an intention-to-treat analysis.

    Measured: Measured at 2 years

Secondary outcomes (16)

  1. Neutrophil Engraftment

    Measured: Measured at Day 28

  2. Platelet Engraftment

    Measured: Measured at Day 180

  3. Graft Failure

    Measured: Measured at 28 and 100 days

  4. Extensive Chronic Graft-versus-host Disease (GVHD)

    Measured: Measured at 730 days

  5. Chronic GVHD

    Measured: Measured at 2 years

  6. Relapse

    Analysis restricted to patients who received the transplant.

    Measured: Measured at 2 years

  7. Infections

    Number of infection reports per patient.

    Measured: Measured at 1 and 2 years

  8. Grades III-V Unexpected Adverse Events

    Measured: Measured by 2 years

  9. Acute GVHD Grade II-IV

    Measured: 100 days, 180 days

  10. Acute GVHD Grade III-IV

    Measured: 100 days, 180 days

  11. Current Immunosuppressive (IS) Free Survival

    This outcome measure takes into account subsequent immunosuppressive therapy that may occur following discontinuation of initial immunosuppressive therapy.

    Measured: Measured at 2 years

  12. Immune Reconstitution

    Measured: Measured at 100 days, 6 months, and 1 and 2 years

  13. Donor Recovery of Baseline Complete Blood Count (CBC) and White Blood Cell Count (WBC) Differential

    Measured: Measured at 1, 6, and 12 months

  14. Donor Recovery to Baseline Toxicity Scores

    Measured: Measured at 1, 6, and 12 months

  15. Donor Quality of Life

    Measured: Measured at 1, 6, and 12 months

  16. Patient Quality of Life

    Measured: Measured at baseline, 6 months, and 1, 2, and 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Jan 2004 (estimated)
  2. Study startJan 2004
  3. Primary completionApr 2013
  4. Study completionApr 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
551 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00075816
Sponsor's protocol ID
BMTCTN0201
Other IDs
U01HL069294-05, BMT CTN 0201, 5U24CA076518
Status last verified
Dec 2022
First submitted
9 Jan 2004
Last updated
4 Jan 2023
Data monitoring committee
Yes
Individual data sharing
Yes: Results will be published in a manuscript

Publications

  • Anasetti C, Logan BR, Lee SJ, Waller EK, Weisdorf DJ, Wingard JR, Cutler CS, Westervelt P, Woolfrey A, Couban S, Ehninger G, Johnston L, Maziarz RT, Pulsipher MA, Porter DL, Mineishi S, McCarty JM, Khan SP, Anderlini P, Bensinger WI, Leitman SF, Rowley SD, Bredeson C, Carter SL, Horowitz MM, Confer DL; Blood and Marrow Transplant Clinical Trials Network. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012 Oct 18;367(16):1487-96. doi: 10.1056/NEJMoa1203517.

    PubMed 23075175

  • Foley B, Cooley S, Verneris MR, Curtsinger J, Luo X, Waller EK, Anasetti C, Weisdorf D, Miller JS. Human cytomegalovirus (CMV)-induced memory-like NKG2C(+) NK cells are transplantable and expand in vivo in response to recipient CMV antigen. J Immunol. 2012 Nov 15;189(10):5082-8. doi: 10.4049/jimmunol.1201964. Epub 2012 Oct 17.

    PubMed 23077239

  • Switzer GE, Bruce JG, Harrington D, Haagenson M, Drexler R, Foley A, Confer D, Bishop M, Anderlini P, Rowley S, Leitman SF, Anasetti C, Wingard JR. Health-related quality of life of bone marrow versus peripheral blood stem cell donors: a prespecified subgroup analysis from a phase III RCT-BMTCTN protocol 0201. Biol Blood Marrow Transplant. 2014 Jan;20(1):118-27. doi: 10.1016/j.bbmt.2013.10.024. Epub 2013 Nov 1.

    PubMed 24184336

  • Waller EK, Logan BR, Harris WA, Devine SM, Porter DL, Mineishi S, McCarty JM, Gonzalez CE, Spitzer TR, Krijanovski OI, Linenberger ML, Woolfrey A, Howard A, Wu J, Confer DL, Anasetti C. Improved survival after transplantation of more donor plasmacytoid dendritic or naive T cells from unrelated-donor marrow grafts: results from BMTCTN 0201. J Clin Oncol. 2014 Aug 1;32(22):2365-72. doi: 10.1200/JCO.2013.54.4577. Epub 2014 Jun 30.

    PubMed 24982459

  • Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ. Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial. Biol Blood Marrow Transplant. 2015 Oct;21(10):1815-22. doi: 10.1016/j.bbmt.2015.06.004. Epub 2015 Jun 11.

    PubMed 26071866

  • Young JH, Logan BR, Wu J, Wingard JR, Weisdorf DJ, Mudrick C, Knust K, Horowitz MM, Confer DL, Dubberke ER, Pergam SA, Marty FM, Strasfeld LM, Brown JWM, Langston AA, Schuster MG, Kaul DR, Martin SI, Anasetti C; Blood and Marrow Transplant Clinical Trials Network Trial 0201. Infections after Transplantation of Bone Marrow or Peripheral Blood Stem Cells from Unrelated Donors. Biol Blood Marrow Transplant. 2016 Feb;22(2):359-370. doi: 10.1016/j.bbmt.2015.09.013. Epub 2015 Sep 25.

    PubMed 26409243

  • Burns LJ, Logan BR, Chitphakdithai P, Miller JP, Drexler R, Spellman S, Switzer GE, Wingard JR, Anasetti C, Confer DL; Blood and Marrow Transplant Clinical Trials Network. Recovery of Unrelated Donors of Peripheral Blood Stem Cells versus Recovery of Unrelated Donors of Bone Marrow: A Prespecified Analysis from the Phase III Blood and Marrow Transplant Clinical Trials Network Protocol 0201. Biol Blood Marrow Transplant. 2016 Jun;22(6):1108-1116. doi: 10.1016/j.bbmt.2016.02.018. Epub 2016 Mar 21.

    PubMed 27013014

  • Lee SJ, Logan B, Westervelt P, Cutler C, Woolfrey A, Khan SP, Waller EK, Maziarz RT, Wu J, Shaw BE, Confer D, Horowitz MM, Anasetti C. Comparison of Patient-Reported Outcomes in 5-Year Survivors Who Received Bone Marrow vs Peripheral Blood Unrelated Donor Transplantation: Long-term Follow-up of a Randomized Clinical Trial. JAMA Oncol. 2016 Dec 1;2(12):1583-1589. doi: 10.1001/jamaoncol.2016.2520.

    PubMed 27532508

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Allogeneic bone marrow transplantation and Peripheral blood stem cell transplantation in people with Leukemia and Myeloproliferative Disorders, run by Medical College of Wisconsin at 45 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 45 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Feb 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Two-year Overall Survival (time frame: Measured at 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00075816, which also lists 8 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jan 2023.

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