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NCT00445744 · REGISTRY CONDITION SEARCH

Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome

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

Phase
NA
Status at capture
COMPLETED
Registry last update
2018-01-02

This trial is studying the side effects and how well giving cyclophosphamide and busulfan followed by donor stem cell transplant works in treating patients with myelofibrosis, acute myeloid leukemia, or myelodysplastic syndrome. Giving chemotherapy, such as cyclophosphamide and busulfan, before a donor stem cell transplant helps stops the growth of cancer cells. It also helps 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 after the 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

Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity

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outcome · POSTED

Non-relapse Mortality (NRM) (Patients With AML/MDS)

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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: * Idiopathic myelofibrosis (CIMF) * Myelofibrosis developing with polycythemia vera or essential thrombocythemia * Acute myeloid leukemia with or without antecedent hematologic disorder, at any disease stage (complete remission, minimal residual disease, or relapsed leukemia) * Myelodysplastic syndrome of any World Health Organization (WHO) or French-American-British (FAB) category, at any disease stage * Less than 61 years of age if transplanted from an unrelated donor, or less than 66 years of age if transplanted from a related donor * Receiving unmanipulated peripheral blood stem cells from an human leukocyte antigen (HLA)-identical or 1-allele-mismatched related or unrelated donor, or receiving G-CSF-stimulated bone marrow if co-enrolled on Fred Hutchinson Cancer Research Center (FHCRC) protocol 2250 * With a Karnofsky Performance score of \> 70% at the time of pre-transplant evaluation * Able to give informed consent (if \>= 18 years of age), or with a legal guardian capable of giving consent (if \< 18 years of age) * DONOR: HLA-identical or 1-allele-mismatched related or unrelated donors (by high resolution typing) * DONOR: Undergoing peripheral blood stem cell harvest or G-CSF-stimulated bone marrow harvest (bone marrow permitted only as part of FHCRC protocol 2250) * DONOR: In good general health, with a Karnofsky performance score of \> 80% * DONOR: Able to give informed consent (if \>= 18 years of age), or with a legal guardian able to give informed consent (if \< 18 years of age and donating for a related transplant) Exclusion Criteria: * Without an HLA-identical or 1-allele-mismatched related or unrelated donor * With human immunodeficiency virus (HIV) positivity or active infectious hepatitis * Receiving a medication known to strongly inhibit enzymes in the CYP450 pathway, and which, in the judgment of the consenting provider, cannot be safely discontinued for the duration of conditioning * Whose life expectancy is severely limited by diseases other than the hematologic disorder for which they are undergoing HCT (HCT-comorbidity index \[CI\] \> 3) * Women who are pregnant or lactating * With known hypersensitivity to BU or CY * With hepatic dysfunction as evidenced by total bilirubin or AST \> 2x the upper limit of normal, or evidence of synthetic dysfunction or cirrhosis * With impaired renal function, as evidenced by creatinine clearance \< 50% of expected, creatinine \> 2x the upper limit of normal, or dialysis dependence * With impaired pulmonary function, as evidenced by pO2 \< 70 mm Hg and diffusing capacity of carbon monoxide (DLCO) \< 70% predicted or by pO2 \< 80 mm Hg and DLCO \< 60%, or receiving continuous supplementary oxygen * With impaired cardiac function, as evidenced by ejection fraction \< 35% or active coronary artery disease * Unable to give informed consent * DONOR: Deemed unable to undergo stem cell collection, for any reason * DONOR: HIV-positive, or hepatitis B or C antigen-positive * DONOR: Women with a positive pregnancy test * DONOR: Unable to give informed consent (if \>= 18 years of age), or without a legal guardian able to give informed consent (if \<18 years of age)
healthy Volunteers
false
maximum Age
65 Years
sex
ALL
std Ages
  1. CHILD
  2. ADULT
  3. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11.
    intervention Names
    1. Drug: cyclophosphamide
    2. Drug: busulfan
    3. Drug: tacrolimus
    4. Drug: methotrexate
    5. Genetic: cytogenetic analysis
    6. Other: flow cytometry
    7. Other: pharmacological study
    8. Other: pharmacogenomic studies
    9. Procedure: peripheral blood stem cell transplantation
    10. Procedure: allogeneic hematopoietic stem cell transplantation
    label
    Treatment (cyclophosphamide, busulfan, transplant)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Given IV
    name
    cyclophosphamide
    other Names
    1. CPM
    2. CTX
    3. Cytoxan
    4. Endoxan
    5. Endoxana
    type
    DRUG
  2. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Given IV
    name
    busulfan
    other Names
    1. BSF
    2. BU
    3. Misulfan
    4. Mitosan
    5. Myeloleukon
    type
    DRUG
  3. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Given IV or PO
    name
    tacrolimus
    other Names
    1. FK 506
    2. Prograf
    type
    DRUG
  4. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Given IV
    name
    methotrexate
    other Names
    1. amethopterin
    2. Folex
    3. methylaminopterin
    4. Mexate
    5. MTX
    type
    DRUG
  5. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Correlative studies
    name
    cytogenetic analysis
    type
    GENETIC
  6. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Correlative studies
    name
    flow cytometry
    type
    OTHER
  7. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Correlative studies
    name
    pharmacological study
    other Names
    1. pharmacological studies
    type
    OTHER
  8. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Correlative studies
    name
    pharmacogenomic studies
    other Names
    1. Pharmacogenomic Study
    type
    OTHER
  9. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Undergo PBPC transplantation
    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
  10. arm Group Labels
    1. Treatment (cyclophosphamide, busulfan, transplant)
    description
    Undergo allogeneic transplantation
    name
    allogeneic hematopoietic stem cell transplantation
    type
    PROCEDURE
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
52
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Number of patients with regimen-related liver toxicity. Diagnoses will be made according to the established criteria initially proposed in 1984 by McDonald et al.
    measure
    Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity
    time Frame
    Up to day +20
  2. description
    Cumulative incidence rate with death as a competing risk, assessed at day 100.
    measure
    Non-relapse Mortality (NRM) (Patients With AML/MDS)
    time Frame
    Up to day 200
Full study description
brief Summary
This trial is studying the side effects and how well giving cyclophosphamide and busulfan followed by donor stem cell transplant works in treating patients with myelofibrosis, acute myeloid leukemia, or myelodysplastic syndrome. Giving chemotherapy, such as cyclophosphamide and busulfan, before a donor stem cell transplant helps stops the growth of cancer cells. It also helps 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 after the transplant may stop this from happening
detailed Description
PRIMARY OBJECTIVES: I. To estimate the incidence of hepatotoxicity with a conditioning regimen of CY (cyclophosphamide)/tBU (busulfan) in patients receiving hematopoietic cell transplant (HCT). SECONDARY OBJECTIVES: I. To determine overall and non-relapse mortality at day +200 after HCT. II. To determine the peak bilirubin levels through day +20 after HCT. III. To determine the incidence of pulmonary toxicity in the form of idiopathic pulmonary syndrome (IPS). IV. To determine the rate of graft failure. V. To determine the time to engraftment. VI. To determine the rate of relapse. VII. To determine the incidence and severity of graft-versus-host disease (GVHD). VIII. To evaluate the pharmacokinetics/dynamics of BU and CY. X. To evaluate the pharmacogenomics of response, toxicity and pharmacokinetics of CY/tBU. OUTLINE: CONDITIONING REGIMEN: Patients receive cyclophosphamide intravenously (IV) on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or orally (PO) twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. After completion of study treatment, patients are followed periodically.
Source references
Source notices and limitations
    Discovery and provenance
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      query cond
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    Preserved source evidence · Not medical advice