Stem Cell Transplantation for Hematological Malignancies

This study has been terminated.
(Replaced by a different study)
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00176839
First received: September 12, 2005
Last updated: March 12, 2013
Last verified: March 2013
  Purpose

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.


Condition Intervention Phase
Leukemia, Lymphocytic, Acute
AML
MDS
Procedure: Stem Cell Transplant
Drug: Busulfan
Drug: Cyclophosphamide
Drug: Melphalan
Drug: G-CSF
Drug: ATG
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Busulfan, Cyclophosphamide, and Melphalan Followed by Allogeneic Hematopoietic Cell Transplantation in Patients With Hematological Malignancies

Resource links provided by NLM:


Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:
  • Probability of Long-term Disease-free Survival (DFS) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    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 [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Number of patients with relapse after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.


Enrollment: 11
Study Start Date: June 2000
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment Arm
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.
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.
Other Names:
  • Bone Marrow Transplant
  • umbilical cord transplant
  • hematopoietic stem cell transplant
Drug: Busulfan
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, four times a day for four days (days -7 through -4).
Other Name: Busulfex, Myleran
Drug: Cyclophosphamide
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.
Other Name: Cytoxan, Neosar
Drug: Melphalan
MELPHALAN will be given via the central venous line for one day, on day -1, prior to stem cell transplantation.
Other Name: Alkeran
Drug: G-CSF
G-CSF is to be given daily IV beginning on day +1 until ANC 2.5 x 109/L.
Other Name: granulocyte colony-stimulating factor, Filgrastim, Neupogen
Drug: ATG
ATG will be administered to umbilical cord blood recipients.
Other Name: antithymocyte globulin

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.

  Eligibility

Ages Eligible for Study:   up to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00176839

Locations
United States, Minnesota
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Margaret MacMillan, MD Masonic Cancer Center, University of Minnesota
  More Information

No publications provided

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176839     History of Changes
Other Study ID Numbers: 2000LS040, MT2000-12, 0005M52481
Study First Received: September 12, 2005
Results First Received: January 11, 2013
Last Updated: March 12, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Masonic Cancer Center, University of Minnesota:
Stem Cell transplant
retransplant
hematological malignancies

Additional relevant MeSH terms:
Neoplasms
Leukemia, Lymphoid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Cyclophosphamide
Melphalan
Busulfan
Antilymphocyte Serum
Lenograstim
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Adjuvants, Immunologic

ClinicalTrials.gov processed this record on October 16, 2014