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Stem Cell Transplant for Hematological Malignancy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00176930
Recruitment Status : Terminated (replaced my another study ; Trial re-written as MT2015-29)
First Posted : September 15, 2005
Last Update Posted : January 9, 2020
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Brief Summary:

The purpose of this study is to develop a standard of care treatment using allogeneic stem cells for patients with cancers of the blood.

The protocol was revised to reflect that this study is considered "treatment guidelines", rather than a research study.

Condition or disease Intervention/treatment Phase
Leukemia, Myeloid, Chronic AML Leukemia, Lymphocytic, Acute MDS Leukemia, Lymphocytic, Chronic JMML Hodgkin's Disease Non-hodgkin's Lymphoma Multiple Myeloma Procedure: Stem Cell Transplant Drug: Cyclophosphamide Radiation: Total Body Irradiation Drug: Busulfan Not Applicable

Detailed Description:

Preparative regimen using total body irradiation (TBI) and cyclophosphamide:

  1. on day -6 and -5: cyclophosphamide is given,
  2. on day -4, -3, -2, and -1: TBI is given,
  3. on day 0: stem cell or bone marrow is infused.

Alternate preparative therapy for patients not able to receive TBI

The chemotherapy (cyclophosphamide and busulfan) is given with the intent of destroying the bone marrow, eliminating any cancerous and preparing for the transplant of the donor's blood stem cells by suppressing the immune system.

l. Ten days before the transplant (Day 10), subjects will be admitted to the bone marrow transplant unit and placed in isolation to reduce exposure to infections. Isolation will be continued until adequate numbers of cells are present in the blood to fight infection.

2. On day -9, -8, -7, -6 busulfan is given.

3. On day -5, -4, -3, -2 cyclophosphamide is given.

4. On day -1 no therapy is given (day of rest).

5. On day 0 the donor stem cells are given intravenously. Additional cells may be given on day +1 or 2 as needed.


Subjects will be admitted to the bone marrow transplant unit and put in isolation to reduce exposure to infectious agents. During this time, they will receive the preparative treatment outlined above. Once they have received the preparative regimen, stem cells will be obtained from the donor and given intravenously.

The new stem cells will replace the bone marrow that was damaged by the treatment for the cancer.

Isolation will be continued until adequate numbers of cells are present in the blood to fight infection. Subjects will then be transferred from the bone marrow transplant unit and discharged from the hospital when medically ready. Subjects will be expected to return for follow-up to the bone marrow transplant clinic at specific dates as determined by their physician.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 330 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Transplant for Hematological Malignancy
Actual Study Start Date : October 2001
Actual Primary Completion Date : December 2019
Actual Study Completion Date : December 2019

Arm Intervention/treatment
Experimental: Allogeneic stem cell transplant
Patients receiving cyclophosphamide and total body irradiation (TBI) and transplant, or cyclophosphamide, Busulfan and transplant.
Procedure: Stem Cell Transplant
Certain cancers can be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease, such as cancer. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover. (Allowable sources of stem cells = related or unrelated bone marrow or peripheral blood, for Busulfan/cyclophosphamide/ATG preparative chemo only, umbilical cord blood is also permitted.)
Other Name: Bone Marrow Transplant.

Drug: Cyclophosphamide
60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.
Other Name: Cytoxan

Radiation: Total Body Irradiation
On Day -4, -3, -2, -1 total body irradiation is given twice daily.
Other Name: Radiation therapy

Drug: Busulfan
When not receiving total body irradiation, administered Days -9 through -6, 0.8 mg/kg/dose by intravenous dosing every 6 hours.
Other Names:
  • Busulfex
  • Myleran

Primary Outcome Measures :
  1. Disease-Free Survival [ Time Frame: Long-term (1 and 2 year) ]
    is the length of time during and after medication or treatment during which the disease being treated (usually cancer) does not get worse. It is sometimes used as a metric to study the health of a person with a disease to try to determine how well a new treatment is working.

Secondary Outcome Measures :
  1. Time to Neutrophil Engraftment [ Time Frame: Day 42 ]
    Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.

  2. Incidence of Acute Graft-Versus-Host Disease [ Time Frame: Day 100 ]
    Acute Graft-Versus-Host Disease (aGVHD) is a severe short-term complication created by infusion of donor cells into a foreign host.

  3. Incidence of Chronic GVHD [ Time Frame: After Day 100 ]
    Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.

  4. Persistence or relapse of malignancy [ Time Frame: 1 year ]
    Defined as the return of disease after its apparent recovery/cessation.

  5. Overall Survival [ Time Frame: 1 year, 2 years ]
    The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer.

  6. Engraftment Failure [ Time Frame: Day 42 ]
    Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Donor will be <75 years of age and in good health.
  • Recipients will be < or = 55 years, will have normal organ function (excluding bone marrow) and will have a Karnofsky activity assessment > or = 90%.
  • Recipients with related or unrelated donor matched at the HLA A, B, DRB1 loci, or mismatched related or unrelated (if < 35 years old) at a single HLA A, B, DRB1 locus.
  • Recipients will be eligible in one of the following disease categories
  • Chronic myelogenous leukemia in accelerated phase or in post blast crisis second or greater chronic phase; or in chronic phase but intolerant of or resistant to tyrosine kinase inhibitors.
  • Acute myelocytic leukemia in first or greater remission, or first, second or third relapse.
  • Acute lymphocytic leukemia in the 2nd or greater bone marrow remission.
  • High risk children will be transplanted in first remission if they meet criteria
  • Myelodysplastic syndrome.
  • Myeloproliferative Diseases - (i.e. myelofibrosis, chronic myelomonocytic leukemia (CMML))
  • Juvenile myelomonocytic leukemia
  • Chronic lymphocytic leukemia
  • Advanced non-Hodgkin's (NHL).
  • Advanced Hodgkin's disease beyond PR2 (> CR3, > PR3).
  • Multiple Myeloma after initial therapy.
  • Donors and recipients signed informed consent

Exclusion Criteria

donors and recipients should meet the following test criteria.

  • required for donors:

    • anti-HIV, Hepatitis B, surface antigen, anti-HCV, CMV, HSV, EBV serologies, pre-priming.
    • CBC, platelet count each day of apheresis, day 0 (or 1 or 2 as needed)
  • required for recipients:

    • anti-HIV, Hepatitis B, surface antigen, anti-HCV, CMV, HSV, EBV serologies, pre-transplant.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00176930

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United States, Minnesota
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
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Principal Investigator: Daniel Weisdorf, MD Masonic Cancer Center, University of Minnesota
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Responsible Party: Masonic Cancer Center, University of Minnesota Identifier: NCT00176930    
Obsolete Identifiers: NCT00393133
Other Study ID Numbers: 2001LS049
MT2001-02 ( Other Identifier: Blood and Marrow Transplantation Program )
0107M05202 ( Other Identifier: IRB, University of Minnesota )
First Posted: September 15, 2005    Key Record Dates
Last Update Posted: January 9, 2020
Last Verified: January 2019
Keywords provided by Masonic Cancer Center, University of Minnesota:
stem cell transplant
chronic leukemia
acute leukemia
Additional relevant MeSH terms:
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Multiple Myeloma
Hematologic Neoplasms
Hodgkin Disease
Leukemia, Myeloid
Leukemia, Lymphoid
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Neoplasms by Histologic Type
Neoplasms, Plasma Cell
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Lymphatic Diseases
Neoplasms by Site
Leukemia, B-Cell
Myeloproliferative Disorders
Bone Marrow Diseases