Skip to content
Triangle logo TriangleGet started
← Study library

NCT00322101 · REGISTRY CONDITION SEARCH

Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia

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

Phase
PHASE3
Status at capture
COMPLETED
Registry last update
2014-10-31

RATIONALE: Giving chemotherapy, such as fludarabine phosphate, busulfan, and cyclophosphamide, and total-body radiation therapy before a donor peripheral 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. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether low-dose chemotherapy and total-body radiation therapy is more effective than high-dose chemotherapy in treating patients with myelodysplastic syndrome or acute myeloid leukemia. PURPOSE: This phase III trial is studying low-dose conditioning to see how well it works compared to high-dose conditioning followed by peripheral blood stem cell transplant in treating patients with myelodysplastic syndromes or acute myeloid leukemia

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

→

baseline

Baseline characteristics

→

outcome · POSTED

Overall Survival

→

outcome · POSTED

Progression-free Survival

→

outcome · POSTED

Non-relapse Mortality

→

outcome · POSTED

Donor Cell Engraftment

→

outcome · POSTED

Incidence of Disease Progression/Relapse

→

outcome · POSTED

Incidence and Severity of Acute and Chronic Graft-vs-host Disease

→

adverse events

Adverse events

→

more info

Limitations, agreements, and source contact

→
Who could take part
eligibility Criteria
Inclusion Criteria: * Myelodysplastic syndrome (MDS) or transformed acute myelogenous leukemia (transformed from MDS) * De novo acute myelogenous leukemia (AML) beyond first remission * Intermediate or high risk de novo AML in first complete response (at FHCRC unrelated donor recipients only) * Chemotherapy required prior to HCT for all patients: * A) Interval between start of a cycle of cytoreductive chemotherapy and infusion of donor stem cells must be at least 30 days; chemotherapy received for disease maintenance will be allowed during this time period * B) All patients must have \< 5% myeloblasts based on marrow morphology performed within 21 days prior to start of conditioning regimen and at least 3-4 weeks after the start of pre-transplant cytoreductive chemotherapy * C) All patients must have no circulating peripheral blood myeloblasts present based on morphologic analysis * Age 65 years or under for patients with related donors; age 60 years or under for patients with unrelated donors * HCT-Specific Comorbidity Index Score (HCT-CI) \< 3 * Related donor (age \> 12 years, nonsyngeneic) or unrelated donor, HLA phenotypically or genotypically identical at the allele level at A,B,C,DRQ1, and CBQ1 * DONOR: Related or unrelated donors who are genotypically or phenotypically matched by high resolution HLA typing (HLA-A, B, C, DRB1, and DQB1); class 1 single allele mismatch allowed * DONOR: Patient and donor pairs homozygous at a mismatched allele in the graft rejection vector are considered a two-allele mismatch, i.e., the patient is A\*0101 and the donor is A\*0201, and this type of mismatch is not allowed * DONOR: A positive anti-donor cytotoxic crossmatch or flow cytometric assay is an absolute donor exclusion at FHCRC/SCCA * DONOR: Age \>= 12 years * DONOR: Donors must consent to PBSC mobilization with G-CSF and leukaphereses; bone marrow as a source of stem cells will not be allowed * DONOR: Donor must have adequate veins for leukaphereses or agree to placement of central venous catheter (femoral, subclavian) Exclusion Criteria: * HIV seropositivity * Fungal infections with radiographic progression after appropriate therapy for greater than one month * Organ dysfunction * Symptomatic coronary artery disease or ejection fraction \< 35% * DLCO \< 65%, FEV1 \< 65% or receiving supplementary continuous oxygen * Liver function abnormalities: Patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, histology, and the degree of portal hypertension; patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin \> 3 mg/dL, and symptomatic biliary disease will be excluded * Karnofsky Performance Score \< 70 * Lansky-Play Performance Score \< 70 for pediatric patients * Life expectancy severely limited (\< 2 years) by disease other than MDS/AML * Fertile men and women unwilling to use contraceptive techniques during and for 12 months following treatment * Patients with active non-hematological malignancies except: * A) Patients with follicular or low grade lymphoma will be eligible as long as they have not and do not require active treatment for control of their disease * B) Patients with localized non-melanoma skin malignancies * Patients with poorly controlled hypertension who are unable to have blood pressure stabilized below 150/90 mm Hg on standard medication * Females who are pregnant or breastfeeding * Patients with systemic, uncontrolled infections * Active CNS disease as identified by positive CSF cytospin * DONOR: Identical twin * DONOR: Age \< 12 years * DONOR: Pregnancy * DONOR: HIV seropositivity * DONOR: Inability to achieve adequate venous access * DONOR: Known adverse reaction to G-CSF
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: Patients receive fludarabine phosphate IV on days -4 to -2 and undergo low-dose total-body irradiation on day 0. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell (PBSC) infusion on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive cyclosporine every 12 hours on days -3 to 57 with taper on days 57-177 or cyclosporine every 12 hours on days -3 to 100 with taper on days 101-177. Patients also receive oral mycophenolate mofetil every 12 hours on days 0-27 or every 8 hours on days 0-40 with taper on days 41-96.
    intervention Names
    1. Radiation: total-body irradiation
    2. Drug: mycophenolate mofetil
    3. Drug: cyclosporine
    4. Drug: fludarabine phosphate
    5. Procedure: peripheral blood stem cell transplantation
    6. Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation
    7. Other: laboratory biomarker analysis
    8. Genetic: cytogenetic analysis
    9. Other: flow cytometry
    10. Genetic: fluorescence in situ hybridization
    11. Other: pharmacological study
    12. Genetic: polymorphism analysis
    label
    Arm I (Nonmyeloablative regimen)
    type
    EXPERIMENTAL
  2. description
    CONDITIONING: Patients are assigned to 1 of 2 treatment groups. Group A: Patients receive fludarabine IV once daily and oral busulfan four times daily or busulfan IV over 3 hours on days -5 to -2. Group B: Patients receive cyclophosphamide IV over 1-2 hours on days -3 and -2 and oral busulfan four times daily or busulfan IV over 3 hours on days -7 to -4. TRANSPLANTATION: Patients undergo PBSC infusion on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV continuously or orally every 12 hours on days -1 to 56 and taper on days 57-200. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
    intervention Names
    1. Procedure: allogeneic hematopoietic stem cell transplantation
    2. Drug: cyclophosphamide
    3. Drug: busulfan
    4. Drug: fludarabine phosphate
    5. Procedure: peripheral blood stem cell transplantation
    6. Other: laboratory biomarker analysis
    7. Other: flow cytometry
    8. Genetic: fluorescence in situ hybridization
    9. Other: pharmacological study
    10. Genetic: polymorphism analysis
    11. Drug: tacrolimus
    12. Drug: methotrexate
    label
    Arm II (Myeloablative regimen)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    description
    Radiation
    name
    total-body irradiation
    other Names
    1. TBI
    type
    RADIATION
  2. arm Group Labels
    1. Arm II (Myeloablative regimen)
    description
    Undergo allogeneic transplantation
    name
    allogeneic hematopoietic stem cell transplantation
    type
    PROCEDURE
  3. arm Group Labels
    1. Arm II (Myeloablative regimen)
    description
    Given IV
    name
    cyclophosphamide
    other Names
    1. CPM
    2. CTX
    3. Cytoxan
    4. Endoxan
    5. Endoxana
    type
    DRUG
  4. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    description
    Given orally
    name
    mycophenolate mofetil
    other Names
    1. Cellcept
    2. MMF
    type
    DRUG
  5. arm Group Labels
    1. Arm II (Myeloablative regimen)
    description
    Given IV or orally
    name
    busulfan
    other Names
    1. BSF
    2. BU
    3. Misulfan
    4. Mitosan
    5. Myeloleukon
    type
    DRUG
  6. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    description
    Given IV or orally
    name
    cyclosporine
    other Names
    1. ciclosporin
    2. cyclosporin
    3. cyclosporin A
    4. CYSP
    5. Sandimmune
    type
    DRUG
  7. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    2. Arm II (Myeloablative regimen)
    description
    Given IV
    name
    fludarabine phosphate
    other Names
    1. 2-F-ara-AMP
    2. Beneflur
    3. Fludara
    type
    DRUG
  8. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    2. Arm II (Myeloablative regimen)
    description
    Undergo 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
  9. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    description
    Undergo allogeneic transplantation
    name
    nonmyeloablative allogeneic hematopoietic stem cell transplantation
    type
    PROCEDURE
  10. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    2. Arm II (Myeloablative regimen)
    description
    Correlative studies
    name
    laboratory biomarker analysis
    type
    OTHER
  11. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    description
    Correlative studies
    name
    cytogenetic analysis
    type
    GENETIC
  12. arm Group Labels
    1. Arm I (Nonmyeloablative regimen)
    2. Arm II (Myeloablative regimen)
    description
    Correlative studies
    name
    flow cytometry
    type
    OTHER
Study design
allocation
RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
25
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. measure
    Overall Survival
    time Frame
    At 2 years
secondary Outcomes
  1. description
    IWG criteria was used to determine disease progression
    measure
    Progression-free Survival
    time Frame
    After stem cell infusion to date of last follow up.
  2. measure
    Non-relapse Mortality
    time Frame
    At 100 days
  3. description
    Chimerism analysis was performed in patients who recieved nonmyeloablative tranplsnat. In this group the definition of engraftment was a CD3 count greater than 50%. In the myeloablative group, engraftment was defined as an absolute neutrophil count greater than 50%.
    measure
    Donor Cell Engraftment
    time Frame
    After stem cell infusion to day 28
  4. description
    Disease progression/relapse was defined by IWG criteria
    measure
    Incidence of Disease Progression/Relapse
    time Frame
    After stem cell infusion to date of last follow up.
  5. measure
    Incidence and Severity of Acute and Chronic Graft-vs-host Disease
    time Frame
    After transplantation
Full study description
brief Summary
RATIONALE: Giving chemotherapy, such as fludarabine phosphate, busulfan, and cyclophosphamide, and total-body radiation therapy before a donor peripheral 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. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether low-dose chemotherapy and total-body radiation therapy is more effective than high-dose chemotherapy in treating patients with myelodysplastic syndrome or acute myeloid leukemia. PURPOSE: This phase III trial is studying low-dose conditioning to see how well it works compared to high-dose conditioning followed by peripheral blood stem cell transplant in treating patients with myelodysplastic syndromes or acute myeloid leukemia
detailed Description
OBJECTIVES: I. Determine whether the conditioning intensity affects outcomes after HCT in patients with MDS or AML who have \< 5% marrow myeloblasts at the time of HCT. OUTLINE: Patients are randomized to 1 of 2 treatment arms. Arm I (Nonmyeloablative regimen): CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and undergo low-dose total-body irradiation on day 0. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell (PBSC) infusion on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive cyclosporine every 12 hours on days -3 to 57 with taper on days 57-177 or cyclosporine every 12 hours on days -3 to 100 with taper on days 101-177. Patients also receive oral mycophenolate mofetil every 12 hours on days 0-27 or every 8 hours on days 0-40 with taper on days 41-96. Arm II (Myeloablative regimen): CONDITIONING: Patients are assigned to 1 of 2 treatment groups. Group A: Patients receive fludarabine IV once daily and oral busulfan four times daily or busulfan IV over 3 hours on days -5 to -2. Group B: Patients receive cyclophosphamide IV over 1-2 hours on days -3 and -2 and oral busulfan four times daily or busulfan IV over 3 hours on days -7 to -4. TRANSPLANTATION: Patients undergo PBSC infusion on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV continuously or orally every 12 hours on days -1 to 56 and taper on days 57-200. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Treatment in both arms continues in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up periodically.
Source references
Source notices and limitations
    Discovery and provenance
    1. release id
      ctgov-registry-1336d216cbd1bbff780dac70
      edge id
      nct-edge-5d47b07359a7d9a6d68c
      requested nct id
      NCT00322101
      primary nct id
      NCT00322101
      source kind
      ctgov_condition_query
      source record id
      Source null
      source file sha256
      a4be8fb2a7db9247bdfed231ef84ae19b3babbe8d619ebf7403cceb730eae2d4
      payload sha256
      c2f023668f3c4774ef6a64789cf3d63f24c3c295e293fc3304e829c7399a7653
      payload
      clinical indication matching status
      condition_index_match_asserted_by_registry_not_reviewed
      end exclusive
      true
      id
      nct-edge-5d47b07359a7d9a6d68c
      link basis
      returned_by_explicit_condition_and_posted_results_filter_query
      nct id
      NCT00322101
      occurrences
      1. context exact
        NCT00322101
        end
        11
        exact text
        NCT00322101
        start
        0
      offset unit
      unicode_code_points
      query cond
      Childhood Myelodysplastic Neoplasms
      query disease state id
      ds-myelodysplastic-neoplasms-pediatric
      source file
      /Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-childhood-myelodysplastic-neoplasms-000.json
      source file sha256
      a4be8fb2a7db9247bdfed231ef84ae19b3babbe8d619ebf7403cceb730eae2d4
      source json pointer
      /studies/66/protocolSection/identificationModule/nctId
      source kind
      ctgov_condition_query
      source record id
      Source null
      source string basis
      parsed_JSON_string_unmodified
      source string sha256
      b720f408e05ad6968d7612ed38ed3323eb9ae13ed9c084bd72e0af325318b58a

    Preserved source evidence · Not medical advice