NCT00176839

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2000

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 7, 2000

Completed
5.3 years until next milestone

First Submitted

Initial submission to the registry

September 12, 2005

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 15, 2005

Completed
6.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2012

Completed
1 year until next milestone

Results Posted

Study results publicly available

February 13, 2013

Completed
Last Updated

December 5, 2017

Status Verified

December 1, 2017

Enrollment Period

11.7 years

First QC Date

September 12, 2005

Results QC Date

January 11, 2013

Last Update Submit

December 3, 2017

Conditions

Keywords

Stem Cell transplantretransplanthematological malignancies

Outcome Measures

Primary Outcomes (1)

  • 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.

    1 year

Secondary Outcomes (5)

  • Probability of Engraftment

    1 year

  • Incidence of Acute Graft-versus-host Disease (GVHD)

    100 days post-transplant

  • Incidence Chronic Graft-versus-host Disease (GVHD)

    1 year

  • Incidence of Regimen-related Toxicity 100 Days Post Transplant

    100 days post-transplant

  • Incidence of Relapse

    1 year

Study Arms (1)

Treatment Arm

EXPERIMENTAL

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 TransplantDrug: BusulfanDrug: CyclophosphamideDrug: MelphalanDrug: G-CSFDrug: ATG

Interventions

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.

Also known as: Bone Marrow Transplant, umbilical cord transplant, hematopoietic stem cell transplant
Treatment Arm

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

Also known as: Busulfex, Myleran
Treatment Arm

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.

Also known as: Cytoxan, Neosar
Treatment Arm

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

Also known as: Alkeran
Treatment Arm
G-CSFDRUG

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

Also known as: granulocyte colony-stimulating factor, Filgrastim, Neupogen
Treatment Arm
ATGDRUG

ATG will be administered to umbilical cord blood recipients.

Also known as: antithymocyte globulin
Treatment Arm

Eligibility Criteria

AgeUp to 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • Eligible for TBI containing preparative regimen.
  • Active uncontrolled infection within one week of HCT.
  • Pregnant or lactating females.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-LymphomaHematologic Neoplasms

Interventions

Stem Cell TransplantationBone Marrow TransplantationBusulfanCyclophosphamideMelphalanGranulocyte Colony-Stimulating FactorFilgrastimAntilymphocyte Serum

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesNeoplasms by Site

Intervention Hierarchy (Ancestors)

Cell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, OperativeTissue TransplantationButylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhosphoramidesOrganophosphorus CompoundsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and ProteinsColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesProteinsBiological FactorsImmune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex Mixtures

Results Point of Contact

Title
Margaret MacMillan, M.D.
Organization
University of Minnesota Masonic Cancer Center

Study Officials

  • Margaret MacMillan, MD

    Masonic Cancer Center, University of Minnesota

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 12, 2005

First Posted

September 15, 2005

Study Start

June 7, 2000

Primary Completion

February 1, 2012

Study Completion

February 1, 2012

Last Updated

December 5, 2017

Results First Posted

February 13, 2013

Record last verified: 2017-12

Locations