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NCT00860574 · REGISTRY CONDITION SEARCH

Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE2
Status at capture
COMPLETED
Registry last update
2021-06-22

This phase II trial is studying how well giving treosulfan together with fludarabine phosphate and total-body irradiation followed by donor stem cell transplant works in treating patients with high-risk acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia. Giving chemotherapy, such as treosulfan and fludarabine phosphate, and total-body irradiation before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and methotrexate before and after transplant may stop this from happening

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Relapse Incidence

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outcome · POSTED

Non Relapse Mortality (NRM) Incidence

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outcome · POSTED

Non Relapse Mortality Incidence

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outcome · POSTED

Overall Survival (OS)

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outcome · POSTED

Relapse-free Survival

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outcome · POSTED

Incidence of Grades II-IV Acute GVHD

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outcome · POSTED

Incidence of Chronic GVHD

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outcome · POSTED

Median Donor CD3 + T Lymphocyte Chimerism in Peripheral Blood

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * Acute myeloid leukemia (AML): * All AML patients beyond 1st remission; * Intermediate or high risk AML patients (based on South West Oncology Group \[SWOG\] cytogenetic criteria) in 1st complete remission * Myelodysplastic syndrome (MDS) * Other myeloid malignancies as chronic myelogenous leukemia (CML), CML accelerated phase, CML blast crisis, chronic myelomonocytic leukemia (CMML) (to be approved by patient care conference \[PCC\]) * With Karnofsky Index or Lansky Play-Performance Scale \> 70% on pre-transplant evaluation * Able to give informed consent (if \> 18 years), or with a legal guardian capable of giving informed consent (if \< 18 years) * Previous autologous or allogeneic HCT is allowed * Donors must be: * Human leukocyte antigen (HLA)-identical related donors or * Unrelated donors matched for HLA-A, B, C, DRB1, and DQB1 defined by high resolution deoxyribonucleic acid (DNA) typing or mismatched for one HLA allele, except for HLA-C where no mismatch is allowed * Able to undergo peripheral blood stem cell collection or bone marrow harvest * In good general health, with a Karnofsky or Lansky Play Performance score \> 90% * Able to give informed consent (if \> 18 years), or with a legal guardian capable of giving informed consent (if \< 18 years) * Acute lymphoblastic leukemia (ALL): all ALL patients not eligible for other protocols Exclusion Criteria: * Receiving umbilical cord blood * With impaired cardiac function as evidenced by ejection fraction \< 35% or cardiac insufficiency requiring treatment or symptomatic coronary artery disease * With impaired pulmonary function as evidenced by partial pressure of oxygen (pO2) \< 70 mm Hg and diffusing capacity of the lung for carbon monoxide (DLCO) \< 70% of predicted or pO2 \< 80 mm Hg and DLCO \< 60% of predicted; or receiving supplementary continuous oxygen * With impaired renal function as evidenced by creatinine-clearance \< 50% for age, weight, height or serum creatinine \> 2x upper normal limit or dialysis-dependent * With hepatic dysfunction as evidenced by total bilirubin or aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 2.0 x upper normal limit or evidence of synthetic dysfunction or severe cirrhosis * With active infectious disease requiring deferral of conditioning, as recommended by an Infectious Disease specialist * With human immunodeficiency virus (HIV)-positivity or active infectious hepatitis because of possible risk of lethal infection when treated with immunosuppressive therapy * With central nervous system (CNS) leukemic involvement not clearing with intrathecal chemotherapy and/or cranial radiation prior to initiating conditioning (day -6) * With life expectancy severely limited by diseases other than malignancy * Women who are pregnant or lactating because of possible risk to the fetus or infant * With known hypersensitivity to treosulfan and/or fludarabine * Receiving another experimental drug within 4 weeks before initiation of conditioning (day -6) * Unable to give informed consent (if \> 18 years) or with a legal guardian (if \< 18 years) unable to give informed consent * Ineligible donors will be those: * Deemed unable to undergo marrow harvesting or PBSC mobilization and leukapheresis * Who are HIV-positive * With active infectious hepatitis * Females with a positive pregnancy test * Unable to give informed consent (if \> 18 years) or with a legal guardian (if \< 18 years) unable to give informed consent
healthy Volunteers
false
maximum Age
60 Years
sex
ALL
std Ages
  1. CHILD
  2. ADULT
Treatment arms and interventions
arm Groups
  1. description
    CONDITIONING REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -6 to day -2 and treosulfan IV over 2 hours on days -6 to day -4. Patients also undergo total-body irradiation on day 0. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplantation or bone marrow transplantation on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV continuously or PO BID on days -1 to 56, followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
    intervention Names
    1. Drug: treosulfan
    2. Drug: fludarabine phosphate
    3. Radiation: total-body irradiation
    4. Procedure: peripheral blood stem cell transplantation
    5. Drug: tacrolimus
    6. Procedure: allogeneic bone marrow transplantation
    7. Procedure: allogeneic hematopoietic stem cell transplantation
    8. Drug: methotrexate
    label
    Treatment (allogeneic transplantation)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV
    name
    treosulfan
    other Names
    1. dihydroxybusulfan
    2. Ovastat
    3. tresulfon
    type
    DRUG
  2. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV
    name
    fludarabine phosphate
    other Names
    1. 2-F-ara-AMP
    2. Beneflur
    3. Fludara
    type
    DRUG
  3. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Low dose starting at 2Gy
    name
    total-body irradiation
    other Names
    1. TBI
    type
    RADIATION
  4. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV per institutional standard practice
    name
    peripheral blood stem cell transplantation
    other Names
    1. PBPC transplantation
    2. PBSC transplantation
    3. peripheral blood progenitor cell transplantation
    4. transplantation, peripheral blood stem cell
    type
    PROCEDURE
  5. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV or PO
    name
    tacrolimus
    other Names
    1. FK 506
    2. Prograf
    type
    DRUG
  6. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV per institutional standard practice
    name
    allogeneic bone marrow transplantation
    other Names
    1. bone marrow therapy, allogeneic
    2. bone marrow therapy, allogenic
    3. transplantation, allogeneic bone marrow
    4. transplantation, allogenic bone marrow
    type
    PROCEDURE
  7. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV per institutional standard practice
    name
    allogeneic hematopoietic stem cell transplantation
    type
    PROCEDURE
  8. arm Group Labels
    1. Treatment (allogeneic transplantation)
    description
    Given IV
    name
    methotrexate
    other Names
    1. amethopterin
    2. Folex
    3. methylaminopterin
    4. Mexate
    5. MTX
    type
    DRUG
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
96
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. measure
    Relapse Incidence
    time Frame
    At 6 months
  2. description
    Cumulative incidence of NRM at 6 months. NRM includes all deaths without relapse or disease progression.
    measure
    Non Relapse Mortality (NRM) Incidence
    time Frame
    At 6 months
secondary Outcomes
  1. measure
    Non Relapse Mortality Incidence
    time Frame
    1 year after HCT
  2. measure
    Overall Survival (OS)
    time Frame
    at 2 years
  3. measure
    Relapse-free Survival
    time Frame
    at 2 years
  4. measure
    Incidence of Grades II-IV Acute GVHD
    time Frame
    at 6 months
  5. measure
    Incidence of Chronic GVHD
    time Frame
    at 6 months
  6. description
    Donor chimerism was evaluated in peripheral blood T cells
    measure
    Median Donor CD3 + T Lymphocyte Chimerism in Peripheral Blood
    time Frame
    Day 28 after HCT
Full study description
brief Summary
This phase II trial is studying how well giving treosulfan together with fludarabine phosphate and total-body irradiation followed by donor stem cell transplant works in treating patients with high-risk acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia. Giving chemotherapy, such as treosulfan and fludarabine phosphate, and total-body irradiation before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and methotrexate before and after transplant may stop this from happening
detailed Description
PRIMARY OBJECTIVES: I. Decrease the incidence of relapse to \< 15% at 6 month post transplant in patients with high risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) transplanted from related or unrelated donors, without unacceptably increasing toxicity (10% non-relapse mortality \[NRM\] at 6 months). SECONDARY OBJECTIVES: I. Evaluate the incidence of NRM at 180 days and 1 year after hematopoietic cell transplantation (HCT). II. Evaluate overall survival (OS) and relapse-free survival (RFS). III. Incidence of grades II-IV acute graft-versus-host disease (GVHD). IV. Incidence of chronic GVHD. V. Donor chimerism on days +28 and +100. OUTLINE: CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -6 to day -2 and treosulfan IV over 2 hours on days -6 to day -4. Patients also undergo total-body irradiation on day 0. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplantation or bone marrow transplantation on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV continuously or orally (PO) twice daily (BID) on days -1 to 56, followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. After completion of study treatment, patients are followed up periodically.
Source references
Source notices and limitations
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