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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.
 
ClinicalTrials.gov Identifier: NCT00176930
Recruitment Status : Terminated (replaced by another study ; Trial re-written as MT2015-29)
First Posted : September 15, 2005
Results First Posted : January 22, 2021
Last Update Posted : January 22, 2021
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Description
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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 Biological: Stem Cell Transplant Drug: Cyclophosphamide Radiation: Total Body Irradiation Drug: Busulfan Drug: Equine ATG (ATGAM) Biological: CD4+/CD25+ cells 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.

Transplant:

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 330 participants
Allocation: Non-Randomized
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: PBSC: No TBI
Patients who are not able to receive Total Body Irradiation (TBI) receives cyclophosphamide, Busulfan and Peripheral Blood Stem Cells (PBSC) as a source of transplant
 Biological: 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

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
Experimental: Marrow : No TBI
Patients who are not able to receive Total Body Irradiation (TBI) receives cyclophosphamide, Busulfan and Bone Marrow as a source of stem cell transplant
 Biological: 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

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
Experimental: UCB : No TBI
Patients who are not able to receive Total Body Irradiation (TBI) receives cyclophosphamide, Busulfan and Umbilical Cord Blood (UCB) as a source of stem cell transplant
 Biological: 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

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
Experimental: UCB : No TBI/Bu/Cy/ATG
Patients who receives Umbilical Cord Blood (UCB) as a source of transplant and who have not had chemotherapy in the prior 3 months receives ATG in addition to cyclophosphamide, Busulfan preparative regimen
 Biological: 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

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

Drug: Equine ATG (ATGAM)
UCB recipients who have not had chemotherapy in the preceding 3 months will also receive Equine ATG (ATGAM) 15 mg/kg IV will be administered every 12 hours for 6 doses beginning on day -3 per institutional guidelines
Experimental: PBSC
Patients receiving cyclophosphamide, Total Body Irradiation (TBI) and Peripheral blood stem cells as a source of transplant
 Biological: 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
Experimental: Marrow
Patients receiving cyclophosphamide, Total Body Irradiation (TBI) and Bone Marrow as a source of stem cell transplant
 Biological: 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
Experimental: UCB
Patients receiving cyclophosphamide, Total Body Irradiation (TBI), and Umbilical Cord Blood (UCB) as a source of stem cell transplant
 Biological: 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
Experimental: Co-Enroll From MT0403
Patients receiving cyclophosphamide, Total Body Irradiation (TBI) , CD4+CD25+ and Peripheral Blood Stem Cells (PBSC) as a source of transplant. These patients are co-enrolled on the MT2004-03 trial (NCT00725062)
 Biological: 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

Biological: CD4+/CD25+ cells
On days -2, patients will receive CD4+/CD25+ cells intravenously.


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants Experiencing Disease-Free Survival at 2 Years Post Transplant [ Time Frame: 2 years ]
    Disease-Free Survival 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.

  2. Number of Participants Experiencing Disease-Free Survival at 5 Years Post Transplant [ Time Frame: 5 years ]
    Disease-Free Survival 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. Number of Participants With 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/mm^3 (0.5 x 10^9/L) or greater.

  2. Number of Participants With Acute Graft-versus-host Disease (GVHD) [ 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. Determine the incidence of grade II-IV acute graft-versus-host disease (GVHD) at day 100 post transplant. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging.

    Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level.


  3. Number of Participants With Chronic Graft-Versus-Host Disease [ Time Frame: 1 year ]
    Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Determine the incidence of chronic GVHD 1 year post transplant. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level.

  4. Number of Participants With Persistence or Relapse of Malignancy at 2 Years Post Transplant [ Time Frame: 2 years ]
    Defined as the return of disease after its apparent recovery/cessation. Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

  5. Number of Participants With Persistence or Relapse of Malignancy at 5 Years Post Transplant [ Time Frame: 5 years ]
    Defined as the return of disease after its apparent recovery/cessation. Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

  6. Number of Participants Who Were Alive at 2 Year Post Transplant [ Time Frame: 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.

  7. Number of Participants Who Were Alive at 5 Year Post Transplant [ Time Frame: 5 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.

  8. Number of Participants Experiencing 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.


Eligibility Criteria
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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
Criteria

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.
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT00176930


Locations
Layout table for location information
United States, Minnesota
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
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Principal Investigator: Daniel Weisdorf, MD Masonic Cancer Center, University of Minnesota
  Study Documents (Full-Text)

Documents provided by Masonic Cancer Center, University of Minnesota:
Study Protocol and Statistical Analysis Plan  [PDF] May 30, 2012

More Information
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov 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
Results First Posted: January 22, 2021
Last Update Posted: January 22, 2021
Last Verified: December 2020
Keywords provided by Masonic Cancer Center, University of Minnesota:
stem cell transplant
chronic leukemia
acute leukemia
irradiation
chemotherapy
Additional relevant MeSH terms:
Layout table for MeSH terms
Leukemia
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
Neoplasms, Plasma Cell
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
 Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Lymphoma
Lymphatic Diseases
Neoplasms by Site
Leukemia, B-Cell
Myeloproliferative Disorders
Bone Marrow Diseases
Cyclophosphamide
Busulfan