Stem Cell Transplantation for Hematological Malignancies

Phase 3TerminatedNCT00176839

Masonic Cancer Center, University of Minnesota11 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Leukemia, Lymphocytic, Acute AML MDS

Interventions

Stem Cell TransplantPROCEDURE

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.

BusulfanDRUG

Prior to transplantation, subjects will receive BUSULFAN via the central venous line, four times a day for four days (days -7 through -4).

CyclophosphamideDRUG

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.

Eligibility

Sex: ALLMax age: 35 Years

Medical Language ↔ Plain English

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.

Locations (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

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.

Time frame: 1 year

Secondary Outcomes

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..

Time frame: 1 year

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.

Time frame: 100 days post-transplant

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.

Time frame: 1 year

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.

Time frame: 100 days post-transplant

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.

Time frame: 1 year

Data from ClinicalTrials.gov

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