Comparison of Peripheral Blood Stem Cell Transplantation With Bone Marrow Transplantation for the Treatment of Serious Hematological Malignancies

This study has been completed.
Sponsor:
Collaborators:
National Marrow Donor Program
University of Minnesota - Clinical and Translational Science Institute
University of Florida
Duke University
Ann & Robert H Lurie Children's Hospital of Chicago
Information provided by:
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00275678
First received: January 10, 2006
Last updated: June 2, 2010
Last verified: June 2010
  Purpose

This study will compare the outcomes of study participants who have had peripheral blood stem cell transplantation or with those who have had bone marrow transplantation from an unrelated donor for the treatment of serious hematological malignancies.


Condition Intervention Phase
Blood Stem Cell Transplant
Procedure: Blood stem cell versus bone marrow transplant
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From HLA Compatible Unrelated Donors.

Resource links provided by NLM:


Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Two-year survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The primary objective is to compare two-year survival probabilities between patients in the two study arms using an intent-to-treat analysis


Secondary Outcome Measures:
  • Two-year survival [ Time Frame: 2 Years ] [ Designated as safety issue: No ]
    Transplanted patients randomized to the two study arms will be compared for the following endpoints: survival, incidences of neutrophil and platelet engraftment, graft failure, acute graft-versus-host disease (GVHD), chronic GVHD, time off all immunosuppressive therapy, relapse, infections, adverse events, immune reconstitution, and quality of life. Donors in each arm of the study will be compared for time to return to baseline toxicity score, CBC and WBC differential values after donation and quality of life.

  • Neutrophil Engraftment > 500/mcl [ Time Frame: 2 Years ] [ Designated as safety issue: No ]
  • Primary graft failure [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Platelet engraftment > 20,000 and 50,000/mcl transfusion independent [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Acute GVHD of grades II-IV and III-IV [ Time Frame: 2 Years ] [ Designated as safety issue: No ]

Enrollment: 15
Study Start Date: November 2003
Primary Completion Date: September 2009 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: Blood stem cell versus bone marrow transplant
    1. Cyclophosphamide and Total Body Irradiation (CY-TBI)-based regimens that include at least 120 mg/kg cyclophosphamide and at least 1200 cGy of fractionated TBI.
    2. Busulfan and cyclophosphamide (BU-CY)-based regimens that include at least 14 mg/kg busulfan orally or 11.2 mg/kg busulfan intravenously (14 x 0.8 correction factor) or a targeted busulfan dosing strategy aimed at a serum concentration greater than 600 ng/mL at steady state and at least 120 mg/kg cyclophosphamide.
    3. Fludarabine and melphalan (Flu-Mel)-based regimens that include a fludarabine dose of least 120 mg per m2 and a melphalan dose of at least 140 mg per m2.
    4. Fludarabine, busulfan, and ATG (Flu-Bu-ATG)-based regimens that include a fludarabine dose of at least 120 mg/m2, at least 8 mg/kg busulfan orally or 250 mg/m2 busulfan intravenously, and at least 40 mg/kg equine ATG or 4 mg/kg rabbit ATG. Institutional standards should be followed for targeting plasma levels.
Detailed Description:

This study is a randomized, open-label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells verses marrow from unrelated donors for transplantation in patients with hematologic malignancies. The study will be comparing two-year survival probabilities between process has many steps after a matched donor is found. The donor also needs to give permission to join this study. Participants will be randomized to either peripheral blood stem cells (PBSC) or bone marrow. The kind of transplant the participant receives is random. Neither the participant nor the doctor may choose the type of transplant.

Participants will receive vaccinations for diphtheria, tetanus, hepatitis B and pneumococcus. Blood samples will be drawn as part of a participant's normal care. Blood samples will also be collected to see if infection fighting cells are working. These samples will be collected at 12 weeks, one year and then yearly until three years post transplant. Information will be collected from the participant's medical history. Participants will also have phone interviews conducted and questions will be asked about their physical and emotional health. Participants taking part in this study may be followed for up to 5 years.

  Eligibility

Ages Eligible for Study:   18 Years to 66 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Participants may be eligible if they are between the ages of 18 to 66 years and are undergoing unrelated blood stem cell transplantation for the first time for the treatment of their hematological malignancy. Additional eligibility criteria will be gathered and evaluated by the transplant physician to determine if an individual is eligible to take part.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00275678

Locations
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
National Marrow Donor Program
University of Minnesota - Clinical and Translational Science Institute
University of Florida
Duke University
Ann & Robert H Lurie Children's Hospital of Chicago
Investigators
Principal Investigator: Shakila P. Khan, M.D. Mayo Clinic
  More Information

No publications provided

Responsible Party: Shakila Khan, MD, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00275678     History of Changes
Other Study ID Numbers: 2158-03
Study First Received: January 10, 2006
Last Updated: June 2, 2010
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Hematologic Diseases

ClinicalTrials.gov processed this record on August 28, 2014