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

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ClinicalTrials.gov Identifier: NCT00450450
Recruitment Status : Active, not recruiting
First Posted : March 22, 2007
Results First Posted : May 9, 2017
Last Update Posted : June 8, 2021
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Study Description
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Brief Summary:
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 or disease Intervention/treatment 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

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Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Study Start Date : December 2007
Actual Primary Completion Date : June 2013

Resource links provided by the National Library of Medicine

Drug Information available for: Filgrastim

Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Estimated Two-year Event-free Survival (EFS) [ Time Frame: at 2 years ]
    EFS is defined as relapse or treatment-related mortality (TRM). relapse is defined by either morphological or cytogenetic evidence of ALL consistent with pre-transplant features.


Secondary Outcome Measures :
  1. Estimated Graft Failure Rate [ Time Frame: Up to 10 years ]
    Primary graft failure is defined as the failure to achieve an absolute neutrophil count of more than 5000 per cubic millimeter for at least three consecutive days by Day +42.

  2. Estimated Incidence of Grade III-IV Acute Graft-versus-host Disease (aGVHD) [ Time Frame: Up to 3 months ]
    Stage III-IV aGVHD is defined as: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI; OR Stage 4 skin, liver or GI involvement.

  3. Estimated 100-day Transplant Related Mortality (TRM) Percentage [ Time Frame: 100 days ]
    Death in a patient after transplant due to protocol treatment is defined as an TRM.

  4. Estimated Percentage of Chronic Graft-versus-host Disease (cGVHD) [ Time Frame: 18 months post-transplant ]
    cGVHD definition is based on BMT CTN MOP SEPT. 2005; outlined in Protocol Appendix III.

  5. Estimated Median Time to Neutrophil Engraftment [ Time Frame: Up to 10 years ]
    Median Time from transplant to neutrophil engraftment

  6. Estimated Median Length of Initial Hospitalization [ Time Frame: Up to 10 years ]
    Estimated and compared between randomization arms using the Wilcoxon rank-sum test.


Other Outcome Measures:
  1. Immune Reconstitution [ Time Frame: Up to 1 year ]
    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.

  2. Infused Nucleated and CD34+ Cell Doses [ Time Frame: Up to 10 years ]
    Compared using the Wilcoxon rank-sum test.


Eligibility Criteria
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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00450450


Locations
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Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Stephan A. Grupp, MD PhD Children's Oncology Group
More Information
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Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT00450450    
Other Study ID Numbers: ASCT0631
NCI-2009-01069 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
COG-ASCT0631 ( Other Identifier: Children's Oncology Group )
COG-PBMTC-STC051 ( Other Identifier: Children's Oncology Group )
CDR0000532926 ( Other Identifier: Clinical Trials.gov )
U10CA098543 ( U.S. NIH Grant/Contract )
First Posted: March 22, 2007    Key Record Dates
Results First Posted: May 9, 2017
Last Update Posted: June 8, 2021
Last Verified: June 2021
Additional relevant MeSH terms:
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Leukemia
Preleukemia
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid, Chronic-Phase
Leukemia, Myelomonocytic, Juvenile
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Neoplasms by Histologic Type
Neoplasms
 Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Lenograstim
Sargramostim
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs