Clinical trial NCT00176930: Stem Cell Transplant for Hematological Malignancy
Official title: Allogeneic Transplant for Hematological Malignancy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Leukemia, Myeloid, Chronic
AML
Leukemia, Lymphocytic, Acute
MDS
Leukemia, Lymphocytic, Chronic
JMML
Hodgkin's Disease
Non-hodgkin's Lymphoma
Multiple Myeloma
Keywords: stem cell transplant
chronic leukemia
acute leukemia
irradiation
chemotherapy
Lead sponsor: Masonic Cancer Center, University of Minnesota
Sponsor class: OTHER
First posted: 2005-09-15
Enrollment: 330
Sex: ALL
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility 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.

Primary outcomes:
measure: Number of Participants Experiencing Disease-Free Survival at 2 Years Post Transplant
timeFrame: 2 years
measure: Number of Participants Experiencing Disease-Free Survival at 5 Years Post Transplant
timeFrame: 5 years
Secondary outcomes:
measure: Number of Participants With Neutrophil Engraftment
timeFrame: Day 42
measure: Number of Participants With Acute Graft-versus-host Disease (GVHD)
timeFrame: Day 100
measure: Number of Participants With Chronic Graft-Versus-Host Disease
timeFrame: 1 year
measure: Number of Participants With Persistence or Relapse of Malignancy at 2 Years Post Transplant
timeFrame: 2 years
measure: Number of Participants With Persistence or Relapse of Malignancy at 5 Years Post Transplant
timeFrame: 5 years
measure: Number of Participants Who Were Alive at 2 Year Post Transplant
timeFrame: 2 years
measure: Number of Participants Who Were Alive at 5 Year Post Transplant
timeFrame: 5 years
measure: Number of Participants Experiencing Engraftment Failure
timeFrame: Day 42
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: Masonic Cancer Center, University of Minnesota
state: Minnesota
Study officials:
affiliation: Masonic Cancer Center, University of Minnesota
name: Daniel Weisdorf, MD
role: PRINCIPAL_INVESTIGATOR
References: