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Donor Bone Marrow Transplant With or Without G-CSF in Treating Young Patients With Hematologic Cancer or Other Diseases

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT00450450
First received: March 20, 2007
Last updated: February 26, 2014
Last verified: February 2014
  Purpose

This randomized phase III trial is studying donor bone marrow transplant with or without G-CSF to compare how well they work in treating young patients with hematologic cancer or other diseases. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer or abnormal cells. It also helps 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving methotrexate and tacrolimus or cyclosporine before and after transplant may stop this from happening. It is not yet known whether donor bone marrow transplant is more effective with or without G-CSF in treating hematologic cancer or other diseases.


Condition Intervention Phase
Childhood Acute Lymphoblastic Leukemia in Remission
Childhood Acute Myeloid Leukemia in Remission
Childhood Chronic Myelogenous Leukemia
Childhood Myelodysplastic Syndromes
Chronic Phase Chronic Myelogenous Leukemia
de Novo Myelodysplastic Syndromes
Juvenile Myelomonocytic Leukemia
Previously Treated Myelodysplastic Syndromes
Recurrent Childhood Acute Lymphoblastic Leukemia
Secondary Myelodysplastic Syndromes
Procedure: allogeneic bone marrow transplantation
Other: laboratory biomarker analysis
Biological: filgrastim
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase III Randomized Trial of G-CSF Stimulated Bone Marrow vs. Conventional Bone Marrow as a Stem Cell Source In Matched Sibling Donor Transplantation

Resource links provided by NLM:


Further study details as provided by Children's Oncology Group:

Primary Outcome Measures:
  • Event-free survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Analyzed using the stratified log-rank test.


Secondary Outcome Measures:
  • Graft failure rate [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]
    A one-sided Z-test of proportions will be used.

  • Incidence of grade III-IV acute graft-versus-host disease [ Time Frame: Up to 3 months ] [ Designated as safety issue: No ]
    A one-sided Z-test of proportions will be used.

  • Treatment-related mortality [ Time Frame: Day 100 ] [ Designated as safety issue: No ]
    A one-sided Z-test of proportions will be used.

  • Incidence of chronic graft-versus-host disease [ Time Frame: 18 months post-transplant ] [ Designated as safety issue: No ]
    Estimated using the cumulative incidence estimator.

  • Time to neutrophil engraftment [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]
    Compared using the log-rank test.

  • Median length of initial hospitalization [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]
    Estimated and compared between randomization arms using the Wilcoxon rank-sum test.

  • Immune reconstitution [ Time Frame: Up to 1 year ] [ Designated as safety issue: Yes ]
    Summarized graphically. Generalized estimating equation will be used to model the levels as a function of time and randomization assignment and to test the impact of G-CSF stimulation on immune reconstruction.

  • Infused nucleated and CD34+ cell doses [ Time Frame: Up to 10 years ] [ Designated as safety issue: No ]
    Compared using the Wilcoxon rank-sum test.


Enrollment: 27
Study Start Date: December 2007
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm I
Patients undergo filgrastim (G-CSF)-stimulated allogeneic bone marrow transplantation on day 0.
Procedure: allogeneic bone marrow transplantation
Patients undergo allogeneic BMT
Other Names:
  • bone marrow therapy, allogeneic
  • bone marrow therapy, allogenic
  • transplantation, allogeneic bone marrow
  • transplantation, allogenic bone marrow
Other: laboratory biomarker analysis
Correlative studies
Biological: filgrastim
Given IV
Other Names:
  • Granulocyte Colony-Stimulating Factor
  • r-metHuG-CSF
  • G-CSF
  • Neupogen
  • NSC #614629
Active Comparator: Arm II
Patients undergo conventional allogeneic bone marrow transplantation on day 0.
Procedure: allogeneic bone marrow transplantation
Patients undergo allogeneic BMT
Other Names:
  • bone marrow therapy, allogeneic
  • bone marrow therapy, allogenic
  • transplantation, allogeneic bone marrow
  • transplantation, allogenic bone marrow
Other: laboratory biomarker analysis
Correlative studies

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of hematologic cancer or other disease, including any of the following:

    • Chronic myelogenous leukemia in first or second chronic phase
    • Acute lymphoblastic leukemia (ALL), meeting any of the following criteria:

      • Relapsed ALL enrolled on a Children's Oncology Group (COG) relapse clinical trial OR received ≥ 1 round of reinduction therapy (4-6 weeks) and 1 round of intensive consolidation chemotherapy (3-6 weeks)
      • ALL in second complete remission (CR)* after a bone marrow, extramedullary, or combined bone marrow and extramedullary relapse
      • Very high-risk ALL in first CR, defined as any of the following:

        • Philadelphia chromosome-positive ALL
        • Hypodiploidy (< 44 chromosomes)
        • Mixed lineage leukemia rearrangement
        • Induction failure
    • Acute myeloid leukemia in first or second CR

      • Induction therapy must be completed
    • Juvenile myelomonocytic leukemia
    • Myelodysplastic syndromes
  • No clinically evident CNS or extramedullary disease
  • No blasts seen on cerebrospinal fluid cytospin
  • Post-relapse reinduction therapy must be completed
  • Not planning to receive reduced-intensity conditioning regimen
  • Not planning to receive a graft that has undergone T-cell depletion
  • No Down syndrome
  • Matched sibling donor must be available and must be enrolled on ASCT0631D companion study
  • Karnofsky performance status (PS) 60-100% (patients > 16 years of age) OR Lansky PS 60-100% (patients ≤ 16 years of age)
  • AST or ALT < 5 times upper limit of normal for age
  • Bilirubin < 2.5 mg/dL (unless due to Gilbert's syndrome)
  • Creatinine clearance or radioisotope glomerular filtration rate ≥ 70 mL/min OR serum creatinine base on age and/or gender as follows:

    • 0.4 mg/dL (1 month to < 6 months of age)
    • 0.5 mg/dL (6 months to < 1 year of age)
    • 0.6 mg/dL (1 to 2 years of age)
    • 0.8 mg/dL (2 to < 6 years of age)
    • 1.0 mg/dL (6 to < 10 years of age)
    • 1.2 mg/dL (10 to < 13 years of age)
    • 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)
    • 1.7 mg/dL (male) or 1.4 mg/dL (female) (≥ 16 years of age)
  • Shortening fraction ≥ 27% by echocardiogram OR LVEF ≥ 50% by radionuclide angiogram
  • FEV_1, FVC, and DLCO ≥ 60% OR meets the following criteria (for patients unable to cooperate for pulmonary function tests):

    • No evidence of dyspnea at rest
    • No exercise intolerance
    • No requirement for supplemental oxygen therapy
  • Not pregnant or nursing
  • No known HIV
  • No known uncontrolled fungal, bacterial, or viral infections

    • Patients acquiring fungal disease during induction therapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by CT scan
  • No prior allogeneic or autologous stem cell transplantation
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00450450

  Show 21 Study Locations
Sponsors and Collaborators
Children's Oncology Group
Investigators
Principal Investigator: Stephan A. Grupp, MD PhD Children's Oncology Group
  More Information

No publications provided

Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT00450450     History of Changes
Other Study ID Numbers: ASCT0631, NCI-2009-01069, COG-ASCT0631, COG-PBMTC-STC051, CDR0000532926, U10CA098543
Study First Received: March 20, 2007
Last Updated: February 26, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Leukemia
Leukemia, Lymphoid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Leukemia, Myeloid, Chronic-Phase
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Leukemia, Myelomonocytic, Juvenile
Myelodysplastic Syndromes
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Preleukemia
Syndrome
Bone Marrow Diseases
Disease
Hematologic Diseases
Immune System Diseases
Immunoproliferative Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Myeloproliferative Disorders
Neoplasms
Neoplasms by Histologic Type
Pathologic Processes
Precancerous Conditions
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Pharmacologic Actions

ClinicalTrials.gov processed this record on November 25, 2014