Stem Cell Transplant for Hematological Malignancy

NCT ID: NCT00176930

Last Updated: 2021-01-22

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

330 participants

Study Classification

INTERVENTIONAL

Study Start Date

2001-10-31

Study Completion Date

2019-12-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Leukemia, Myeloid, Chronic AML Leukemia, Lymphocytic, Acute MDS Leukemia, Lymphocytic, Chronic JMML Hodgkin's Disease Non-hodgkin's Lymphoma Multiple Myeloma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Busulfan

Intervention Type DRUG

When not receiving total body irradiation, administered Days -9 through -6, 0.8 mg/kg/dose by intravenous dosing every 6 hours.

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

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Busulfan

Intervention Type DRUG

When not receiving total body irradiation, administered Days -9 through -6, 0.8 mg/kg/dose by intravenous dosing every 6 hours.

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

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Busulfan

Intervention Type DRUG

When not receiving total body irradiation, administered Days -9 through -6, 0.8 mg/kg/dose by intravenous dosing every 6 hours.

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

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Busulfan

Intervention Type DRUG

When not receiving total body irradiation, administered Days -9 through -6, 0.8 mg/kg/dose by intravenous dosing every 6 hours.

Equine ATG (ATGAM)

Intervention Type DRUG

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

PBSC

Patients receiving cyclophosphamide, Total Body Irradiation (TBI) and Peripheral blood stem cells as a source of transplant

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Total Body Irradiation

Intervention Type RADIATION

On Day -4, -3, -2, -1 total body irradiation is given twice daily.

Marrow

Patients receiving cyclophosphamide, Total Body Irradiation (TBI) and Bone Marrow as a source of stem cell transplant

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Total Body Irradiation

Intervention Type RADIATION

On Day -4, -3, -2, -1 total body irradiation is given twice daily.

UCB

Patients receiving cyclophosphamide, Total Body Irradiation (TBI), and Umbilical Cord Blood (UCB) as a source of stem cell transplant

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Total Body Irradiation

Intervention Type RADIATION

On Day -4, -3, -2, -1 total body irradiation is given twice daily.

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)

Group Type EXPERIMENTAL

Stem Cell Transplant

Intervention Type BIOLOGICAL

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.)

Cyclophosphamide

Intervention Type DRUG

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Total Body Irradiation

Intervention Type RADIATION

On Day -4, -3, -2, -1 total body irradiation is given twice daily.

CD4+/CD25+ cells

Intervention Type BIOLOGICAL

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

Interventions

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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.)

Intervention Type BIOLOGICAL

Cyclophosphamide

60 mg/kg intravenously (IV) Days -6 and -5 or 50 mg/kg/day IV Days -5 through -2.

Intervention Type DRUG

Total Body Irradiation

On Day -4, -3, -2, -1 total body irradiation is given twice daily.

Intervention Type RADIATION

Busulfan

When not receiving total body irradiation, administered Days -9 through -6, 0.8 mg/kg/dose by intravenous dosing every 6 hours.

Intervention Type 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

Intervention Type DRUG

CD4+/CD25+ cells

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

Intervention Type BIOLOGICAL

Other Intervention Names

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Bone Marrow Transplant. Cytoxan Radiation therapy Busulfex Myleran

Eligibility Criteria

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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.
Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Daniel Weisdorf, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

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Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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MT2001-02

Identifier Type: OTHER

Identifier Source: secondary_id

0107M05202

Identifier Type: OTHER

Identifier Source: secondary_id

2001LS049

Identifier Type: -

Identifier Source: org_study_id

NCT00393133

Identifier Type: -

Identifier Source: nct_alias

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