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Stem Cell Transplantation for Hematological Malignancies

Primary Purpose

Leukemia, Lymphocytic, Acute, AML, MDS

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Stem Cell Transplant
Busulfan
Cyclophosphamide
Melphalan
G-CSF
ATG
Sponsored by
Masonic Cancer Center, University of Minnesota
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leukemia, Lymphocytic, Acute focused on measuring Stem Cell transplant, retransplant, hematological malignancies

Eligibility Criteria

undefined - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patients must have a diagnosis of acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and currently be in complete remission. Patients must be either: - <18 years of age who are at least 6 months after initial hematopoietic cell transplant (HCT), - 19-35 years of age and at least 18 months after initial HCT, or - <35 years of age and have received sufficient radiation treatment to be ineligible for total body irradiation (TBI) containing preparative therapy Adequate major organ function including: - Cardiac: ejection fraction > or = 45% - Renal: creatinine clearance > or = 40 mL/min - Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites) - Karnofsky performance status > or = 70% or Lansky score > or = 50% Women of child bearing age must be using adequate birth control and have a negative pregnancy test. Written informed consent. Exclusion Criteria: Eligible for TBI containing preparative regimen. Active uncontrolled infection within one week of HCT. Pregnant or lactating females.

Sites / Locations

  • Masonic Cancer Center, University of Minnesota

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment Arm

Arm Description

Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.

Outcomes

Primary Outcome Measures

Probability of Long-term Disease-free Survival (DFS)
Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Secondary Outcome Measures

Probability of Engraftment
Number of participants with engraftment after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies..
Incidence of Acute Graft-versus-host Disease (GVHD)
Number of participants with acute GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Incidence Chronic Graft-versus-host Disease (GVHD)
Number of participants with chronic GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Incidence of Regimen-related Toxicity 100 Days Post Transplant
Number of participants with regimen-related toxicity 100 days post transplant after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Incidence of Relapse
Number of patients with relapse after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Full Information

First Posted
September 12, 2005
Last Updated
December 3, 2017
Sponsor
Masonic Cancer Center, University of Minnesota
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1. Study Identification

Unique Protocol Identification Number
NCT00176839
Brief Title
Stem Cell Transplantation for Hematological Malignancies
Official Title
Busulfan, Cyclophosphamide, and Melphalan Followed by Allogeneic Hematopoietic Cell Transplantation in Patients With Hematological Malignancies
Study Type
Interventional

2. Study Status

Record Verification Date
December 2017
Overall Recruitment Status
Terminated
Why Stopped
Replaced by a different study
Study Start Date
June 7, 2000 (Actual)
Primary Completion Date
February 2012 (Actual)
Study Completion Date
February 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Masonic Cancer Center, University of Minnesota

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This protocol using busulfan, cyclophosphamide and melphalan has been designed as conditioning therapy for patients receiving stem cell transplantation for acute leukemia or myelodysplastic syndrome (MDS). The hypothesis is that this new regimen will be well tolerated and will cure the patient.
Detailed Description
Subjects will be admitted to the bone marrow transplant unit and put in isolation to reduce exposure to infectious agents. Prior to transplantation, they will receive BUSULFAN via the central venous line, four times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's cancer. As well, these drugs will destroy their immune system to help ensure the new stem cells take and grow after transplantation. On the day of transplantation, umbilical cord blood from the donor will be transfused via venous line. These new cells will replace the subject's bone marrow. After transplantation, the subjects will receive Cyclosporin A and either MMF or MTX Isolation will be continued until adequate numbers of cells are present in the blood to fight infection. Subjects will be discharged from the hospital when medically ready. They will be expected to return for follow-up to the blood and marrow transplant clinic at specific dates as determined by physicians.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukemia, Lymphocytic, Acute, AML, MDS
Keywords
Stem Cell transplant, retransplant, hematological malignancies

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment Arm
Arm Type
Experimental
Arm Description
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
Intervention Type
Procedure
Intervention Name(s)
Stem Cell Transplant
Other Intervention Name(s)
Bone Marrow Transplant, umbilical cord transplant, hematopoietic stem cell transplant
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Busulfan
Other Intervention Name(s)
Busulfex, Myleran
Intervention Description
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, four times a day for four days (days -7 through -4).
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Other Intervention Name(s)
Cytoxan, Neosar
Intervention Description
Prior to stem cell transplantation, subjects will receive CYCLOPHOSPHAMIDE via the central venous line once a day for two days on days -3 and -2.
Intervention Type
Drug
Intervention Name(s)
Melphalan
Other Intervention Name(s)
Alkeran
Intervention Description
MELPHALAN will be given via the central venous line for one day, on day -1, prior to stem cell transplantation.
Intervention Type
Drug
Intervention Name(s)
G-CSF
Other Intervention Name(s)
granulocyte colony-stimulating factor, Filgrastim, Neupogen
Intervention Description
G-CSF is to be given daily IV beginning on day +1 until ANC 2.5 x 109/L.
Intervention Type
Drug
Intervention Name(s)
ATG
Other Intervention Name(s)
antithymocyte globulin
Intervention Description
ATG will be administered to umbilical cord blood recipients.
Primary Outcome Measure Information:
Title
Probability of Long-term Disease-free Survival (DFS)
Description
Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Probability of Engraftment
Description
Number of participants with engraftment after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies..
Time Frame
1 year
Title
Incidence of Acute Graft-versus-host Disease (GVHD)
Description
Number of participants with acute GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Time Frame
100 days post-transplant
Title
Incidence Chronic Graft-versus-host Disease (GVHD)
Description
Number of participants with chronic GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Time Frame
1 year
Title
Incidence of Regimen-related Toxicity 100 Days Post Transplant
Description
Number of participants with regimen-related toxicity 100 days post transplant after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Time Frame
100 days post-transplant
Title
Incidence of Relapse
Description
Number of patients with relapse after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Time Frame
1 year

10. Eligibility

Sex
All
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients must have a diagnosis of acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and currently be in complete remission. Patients must be either: - <18 years of age who are at least 6 months after initial hematopoietic cell transplant (HCT), - 19-35 years of age and at least 18 months after initial HCT, or - <35 years of age and have received sufficient radiation treatment to be ineligible for total body irradiation (TBI) containing preparative therapy Adequate major organ function including: - Cardiac: ejection fraction > or = 45% - Renal: creatinine clearance > or = 40 mL/min - Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites) - Karnofsky performance status > or = 70% or Lansky score > or = 50% Women of child bearing age must be using adequate birth control and have a negative pregnancy test. Written informed consent. Exclusion Criteria: Eligible for TBI containing preparative regimen. Active uncontrolled infection within one week of HCT. Pregnant or lactating females.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Margaret MacMillan, MD
Organizational Affiliation
Masonic Cancer Center, University of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
Masonic Cancer Center, University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

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Stem Cell Transplantation for Hematological Malignancies

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