Stem Cell Transplantation for Hematological Malignancies

NCT ID: NCT00176839

Last Updated: 2017-12-05

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2000-06-07
• Study Completion Date: 2012-02-29

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.

Conditions

Leukemia, Lymphocytic, Acute; AML; MDS

Keywords

Stem Cell transplant; retransplant; hematological malignancies

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Stem Cell Transplant

Intervention Type: PROCEDURE

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.

Busulfan

Intervention Type: DRUG

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

Cyclophosphamide

Intervention Type: DRUG

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.

Melphalan

Intervention Type: DRUG

MELPHALAN will be given via the central venous line for one day, on day -1, prior to stem cell transplantation.

G-CSF

Intervention Type: DRUG

G-CSF is to be given daily IV beginning on day +1 until ANC 2.5 x 109/L.

ATG

Intervention Type: DRUG

ATG will be administered to umbilical cord blood recipients.

Interventions

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.

Intervention Type: PROCEDURE

Busulfan

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

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.

Intervention Type: DRUG

Melphalan

MELPHALAN will be given via the central venous line for one day, on day -1, prior to stem cell transplantation.

Intervention Type: DRUG

G-CSF

G-CSF is to be given daily IV beginning on day +1 until ANC 2.5 x 109/L.

Intervention Type: DRUG

ATG

ATG will be administered to umbilical cord blood recipients.

Intervention Type: DRUG

Other Intervention Names

Bone Marrow Transplant; umbilical cord transplant; hematopoietic stem cell transplant; Busulfex, Myleran; Cytoxan, Neosar; Alkeran; granulocyte colony-stimulating factor, Filgrastim, Neupogen; antithymocyte globulin

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.

• Maximum Eligible Age: 35 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Margaret MacMillan, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

MT2000-12

Identifier Type: OTHER

Identifier Source: secondary_id

0005M52481

Identifier Type: OTHER

Identifier Source: secondary_id

2000LS040

Identifier Type: -

Identifier Source: org_study_id