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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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.

Detailed Summary:

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 mus
Sponsor: Medical College of Wisconsin

Current Primary Outcome: Two-year Overall Survival [ Time Frame: Measured at 2 years ]

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


Original Primary Outcome:

Current Secondary Outcome:

  • Neutrophil Engraftment [ Time Frame: Measured at Day 28 ]
  • Platelet Engraftment [ Time Frame: Measured at Day 180 ]
  • Graft Failure [ Time Frame: Measured at 28 and 100 days ]
  • Extensive Chronic Graft-versus-host Disease (GVHD) [ Time Frame: Measured at 730 days ]
  • Chronic GVHD [ Time Frame: Measured at 2 years ]
  • Relapse [ Time Frame: Measured at 2 years ]
    Analysis restricted to patients who received the transplant.
  • Infections [ Time Frame: Measured at 1 and 2 years ]
    Number of infection reports per patient.
  • Grades III-V Unexpected Adverse Events [ Time Frame: Measured by 2 years ]
  • Acute GVHD Grade II-IV [ Time Frame: 100 days, 180 days ]
  • Acute GVHD Grade III-IV [ Time Frame: 100 days, 180 days ]
  • Current Immunosuppressive (IS) Free Survival [ Time Frame: Measured at 2 years ]
    This outcome measure takes into account subsequent immunosuppressive therapy that may occur following discontinuation of initial immunosuppressive therapy.
  • Immune Reconstitution [ Time Frame: Measured at 100 days, 6 months, and 1 and 2 years ]
  • Donor Recovery of Baseline Complete Blood Count (CBC) and White Blood Cell Count (WBC) Differential [ Time Frame: Measured at 1, 6, and 12 months ]
  • Donor Recovery to Baseline Toxicity Scores [ Time Frame: Measured at 1, 6, and 12 months ]
  • Donor Quality of Life [ Time Frame: Measured at 1, 6, and 12 months ]
  • Patient Quality of Life [ Time Frame: Measured at baseline, 6 months, and 1, 2, and 5 years ]


Original Secondary Outcome:

Information By: Medical College of Wisconsin

Dates:
Date Received: January 9, 2004
Date Started: January 2004
Date Completion:
Last Updated: August 1, 2016
Last Verified: August 2016