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NCT00553202 · REGISTRY CONDITION SEARCH

Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed 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
PHASE2
Status at capture
COMPLETED
Registry last update
2020-04-16

RATIONALE: Giving chemotherapy before a donor stem cell transplant using stem cells that closely match the patient's stem cells, helps stop the growth of cancer cells. It also stops 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 antithymocyte globulin before transplant and cyclosporine, tacrolimus, and methotrexate before and after transplant may stop this from happening. PURPOSE: Natural Killer (NK) cells from the donor's bone marrow may be important in fighting leukemia. Bone marrow donors can be selected based on the type of NK cells they have, specifically the killer immunoglobulin receptor (KIR) type. This study provides information on KIR type from potential donors, which can be used in selecting the bone marrow donor. This phase II trial of unrelated donor stem cell transplant in patients with high risk AML (monosomy 7, -5/5q-, high FLT3-ITD AR, or refractory or relapsed AML) in which KIR typing of the patients and potential donors will be available to the treating transplant physician at the time of donor selection.

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

Overall Survival (OS)

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outcome · POSTED

Cumulative Incidence of NK Cell Reconstitution

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outcome · POSTED

Disease-free Survival

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outcome · POSTED

Acute and Chronic Graft-versus-host Disease

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outcome · POSTED

Time to the Donor-specific NK-cell Receptor Expression

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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
DISEASE CHARACTERISTICS: * Diagnosis of one of the following: * Patients with primary refractory acute myeloid leukemia (AML), defined as ≥ 5% bone marrow blasts after two induction courses of chemotherapy * Primary refractory AML, defined as ≥ 5% bone marrow blasts after two induction courses of chemotherapy * AML or myelodysplastic syndrome with -5/5q- or monosomy 7 without inv(16)/t(16;16) or t(8;21) cytogenetics or NPM or CEBPα mutations * Relapsed AML (≥ 5% bone marrow blasts) who meet the customary WHO criteria for AML * AML and high FLT3 internal tandem duplication allelic ratio (high FLT3-ITD AR), defined as \> 0.4 * All cases of therapy-related AML (therapy-related AML is considered high risk) * Patients with AML, without inv(16)/t(16;16) or t(8;21), monosomy 7, -5/5q-, NPM, or CEPBα mutations, or high FLT3-ITD AR, but with evidence of residual AML (≥ 0.1%) at the end of Induction I; or if a minimal residual disease (MRD) is not performed, then with \> 15% bone marrow blasts by morphology after one induction course of chemotherapy * Any flow-based MRD is eligible for AAML05P1 for patients not on AAML1031, whereas patients on AAML1031 must utilize the central lab as per the AAML1031 protocol guidelines * No Fanconi anemia * Recipients of unrelated marrow or cord blood are eligible for this study PATIENT CHARACTERISTICS: * Karnofsky performance status (PS) (for patients over 16 years of age) or Lansky PS (for patients 16 and under) 50-100% * Total bilirubin ≤ 2 mg/dL * SGOT (AST) or SGPT (ALT) ≤ 2.5 times upper limit of normal * DLCO ≥ 50% OR a normal chest x-ray and pulse oximetry in patients who are unable to undergo pulmonary function tests * Shortening fraction ≥ 27% by ECHO * Creatinine clearance or radioisotope glomerular filtration rate at least 60 mL/min OR creatinine adjusted according to age * HIV negative * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * Patients with proven or suspected bacterial sepsis, pneumonia, or meningitis are eligible provided appropriate therapeutic measures have been initiated to control the presumed or proven infection, and systemic signs are not life-threatening * No evidence or presence of a fungal infection within the past 30 days PRIOR CONCURRENT THERAPY: * Prior chemotherapy, radiotherapy or any antileukemic therapy allowed provided patients meet 1 of the following criteria: * Received initial treatment for relapsed AML * Patients with primary induction failure or relapse who have already received initial therapy and who may have gone on to have additional therapy prior to receiving protocol stipulated therapy on AAML05P1 * No treatment for fungal infection within the past 30 days * Concurrent radiotherapy to localized painful lesions allowed * No other concurrent cancer chemotherapy or immunomodulating agents
healthy Volunteers
false
maximum Age
30 Years
sex
ALL
std Ages
  1. CHILD
  2. ADULT
Treatment arms and interventions
arm Groups
  1. description
    Patients receive busulfan IV every 6 hours on days -9 to -6, high-dose cyclophosphamide IV over 1 hour on days -5 to -2, anti-thymocyte globulin IV once or twice daily over 4 hours on days -3 to -1, and methylprednisolone IV on days -3 to -1. Patients undergo allogeneic hematopoietic stem cell transplantation (SCT) or allogeneic bone marrow transplantation (BMT) on day 0. Patients receive cyclosporine or tacrolimus IV or orally beginning on day -2 and continuing until day 50, followed by a taper until week 24. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Blood samples will be collected periodically from both patients and donors for studies of natural killer cells in support of the pharmacological study objectives
    intervention Names
    1. Biological: anti-thymocyte globulin
    2. Drug: busulfan
    3. Drug: cyclophosphamide
    4. Drug: cyclosporine
    5. Drug: methotrexate
    6. Drug: methylprednisolone
    7. Drug: tacrolimus
    8. Other: laboratory biomarker analysis
    9. Other: pharmacological study
    10. Procedure: allogeneic bone marrow transplantation
    11. Procedure: allogeneic hematopoietic stem cell transplantation
    label
    Treatment (chemotherapy and allogeneic SCT)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV
    name
    anti-thymocyte globulin
    other Names
    1. Rabbit ATG
    2. RATG-Rabbit
    3. Antithymocyte Globulin
    4. Thymoglobulin
    5. NSC #720095
    type
    BIOLOGICAL
  2. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV
    name
    busulfan
    other Names
    1. Busulfex
    2. NSC #750
    type
    DRUG
  3. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV
    name
    cyclophosphamide
    other Names
    1. Cytoxan
    2. NSC #26271
    type
    DRUG
  4. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV or orally
    name
    cyclosporine
    other Names
    1. CYA
    2. Sandimmune
    3. Neoral
    4. Gengraf
    5. NSC #290193
    type
    DRUG
  5. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV
    name
    methotrexate
    other Names
    1. MTX
    2. amethopterin
    3. NSC #000740
    type
    DRUG
  6. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV
    name
    methylprednisolone
    other Names
    1. Solu-Medrol
    2. A-Methapred
    3. Medrol
    4. NSC #19987
    type
    DRUG
  7. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Given IV
    name
    tacrolimus
    other Names
    1. FK-506
    2. Prograf
    3. NSC #717865
    type
    DRUG
  8. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Correlative studies
    name
    laboratory biomarker analysis
    type
    OTHER
  9. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Correlative studies
    name
    pharmacological study
    type
    OTHER
  10. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    allogeneic bone marrow transplantation
    name
    allogeneic bone marrow transplantation
    other Names
    1. bone marrow therapy
    2. allogeneic
    3. allogenic
    4. transplantation
    5. allogeneic bone marrow
    6. allogenic bone marrow
    type
    PROCEDURE
  11. arm Group Labels
    1. Treatment (chemotherapy and allogeneic SCT)
    description
    Undergo allogeneic hematopoietic SCT
    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
158
type
ACTUAL
Registered outcome plans (not posted results)
other Outcomes
  1. description
    The cumulative incidence of relapse or death after SCT will be calculated by considering relapse and death due to other causes as competing events.
    measure
    Disease-free Survival
    time Frame
    From the date of SCT to the date of relapse, the date of death, or the date of last follow-up, whichever occurs first
  2. description
    Acute and chronic GVHD will be summarized.
    measure
    Acute and Chronic Graft-versus-host Disease
    time Frame
    Up to 5 years
  3. description
    The presence of donor cells is demonstrated by the detection of informative variable-number tandem-repeat polymorphisms or by fluorescent in situ hybridization with a Y-chromosome-specific probe in cases of sex-mismatched transplants. Independent variables that will be examined include donor-recipient KIR mismatch, taking into consideration the interactions with donor-recipient human leukocyte antigen (HLA) compatibility, and the numbers of CD34+ cells and CD3+ cells in the graft.
    measure
    Time to the Donor-specific NK-cell Receptor Expression
    time Frame
    Up to 42 days after SCT
primary Outcomes
  1. description
    OS - Time from HSCT until death
    measure
    Overall Survival (OS)
    time Frame
    At 5 years from HSCT date
  2. description
    Cumulative incidence of successful reconstitution to donor level is calculated.
    measure
    Cumulative Incidence of NK Cell Reconstitution
    time Frame
    At 5 years from HSCT date
Full study description
brief Summary
RATIONALE: Giving chemotherapy before a donor stem cell transplant using stem cells that closely match the patient's stem cells, helps stop the growth of cancer cells. It also stops 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 antithymocyte globulin before transplant and cyclosporine, tacrolimus, and methotrexate before and after transplant may stop this from happening. PURPOSE: Natural Killer (NK) cells from the donor's bone marrow may be important in fighting leukemia. Bone marrow donors can be selected based on the type of NK cells they have, specifically the killer immunoglobulin receptor (KIR) type. This study provides information on KIR type from potential donors, which can be used in selecting the bone marrow donor. This phase II trial of unrelated donor stem cell transplant in patients with high risk AML (monosomy 7, -5/5q-, high FLT3-ITD AR, or refractory or relapsed AML) in which KIR typing of the patients and potential donors will be available to the treating transplant physician at the time of donor selection.
detailed Description
OBJECTIVES: * To define the relationship between the status of donor NK-cell receptor and patient outcomes after killer immunoglobulin-like receptor-incompatible unrelated donor (URD) and umbilical cord blood (UCB) hematopoietic cell transplantation (HCT) in young patients with acute myeloid leukemia with monosomy 7, -5/5q-, high FLT3 internal tandem duplication allelic ratio (High-FLT3-ITD AR), or refractory or relapsed acute myelogenous leukemia. * To correlate the relationships between factors affecting NK receptor status and clinical events. * To assess NK-cell development after URD and UCB HCT in patients with poor prognosis AML. * To evaluate NK-cell reconstitution and receptor-acquisition pattern in these patients. OUTLINE: This is a multicenter study. * Preparative regimen: Patients receive 1 of the following regimens: * Hematopoietic stem cell transplantation (SCT): Patients receive busulfan IV every 6 hours on days -9 to -6, high-dose cyclophosphamide IV over 1 hour on days -5 to -2, anti-thymocyte globulin IV once or twice daily over 4 hours on days -3 to -1, and methylprednisolone IV on days -3 to -1. * Umbilical cord blood (UCB) transplantation: Conditioning regimen, infusion procedures, and post-transplant immunoprophylaxis for patients with an UCB donor are according to institutional guidelines and standards. * Allogeneic hematopoietic stem cell transplantation (SCT) or umbilical cord blood (UCB) transplant: Patients undergo allogeneic SCT or UCB transplant on day 0. * Graft-vs-host disease (GVHD) prophylaxis: Patients receive cyclosporine or tacrolimus IV or orally beginning on day -2 and continuing until day 50, followed by a taper until week 24. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Blood samples will be collected periodically from both patients and donors for studies of natural killer cells in support of the study objectives. After completion of study treatment, patients are followed every 6 months for 2 years and then annually for 3 years.
Source references
Source notices and limitations
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    Preserved source evidence · Not medical advice