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Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases

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ClinicalTrials.gov Identifier: NCT00084695
Recruitment Status : Unknown
Verified October 2008 by National Cancer Institute (NCI).
Recruitment status was:  Recruiting
First Posted : June 11, 2004
Last Update Posted : January 10, 2014
Sponsor:
Information provided by:
National Cancer Institute (NCI)

Tracking Information
First Submitted Date  ICMJE June 10, 2004
First Posted Date  ICMJE June 11, 2004
Last Update Posted Date January 10, 2014
Study Start Date  ICMJE September 2003
Estimated Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 17, 2007)
  • Impact of the use of umbilical cord blood as a source of hematopoietic stem cells
  • Comparison of the incidence of graft-vs-host disease with historical data
  • Comparison of the incidence of engraftment with historical data
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00084695 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
Official Title  ICMJE The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells
Brief Summary

RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.

PURPOSE: This phase II trial is studying how well umbilical cord blood works as a source of stem cells in treating patients with types of cancer as well as other diseases.

Detailed Description

OBJECTIVES:

Primary

  • Determine the impact of the use of umbilical cord blood as a source of hematopoietic stem cells for children with life-threatening oncologic, hematologic, or genetic/metabolic disorders in need of a stem cell transplant.
  • Compare the incidence of graft-versus-host disease in patients receiving cord blood transplants in this study with historical data for unrelated donor stem cell transplants.
  • Compare the incidence of engraftment in patients receiving cord blood transplants in this study with historical data for unrelated donor stem cell transplants.

OUTLINE:

  • Preparative therapy: Patients are treated on 1 of 4 preparative therapy regimens.

    • Regimen A: Patients undergo total body irradiation (TBI) two times daily on days -7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and -2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1.
    • Regimen B (patients who do not receive TBI): Patients receive oral busulfan 4 times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV over 15-20 minutes on days -4 to -2.
    • Regimen C (patients with Fanconi's anemia and related disorders): Patients undergo TBI on day -6. Patients receive ATG IV over at least 6 hours and methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
    • Regimen D: Patients receive oral or IV busulfan 4 times daily on days -9 to -5, ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
  • Cord blood transplant: All patients undergo umbilical cord blood transplantation on day 0.
  • Graft-versus-host disease prophylaxis: Patients receive oral or IV cyclosporine twice daily beginning on day -1. Patients also receive methylprednisolone IV twice daily beginning on day 5 and continuing until at least day 28.

PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Childhood Langerhans Cell Histiocytosis
  • Fanconi Anemia
  • Leukemia
  • Lymphoma
  • Myelodysplastic Syndromes
  • Neuroblastoma
  • Sarcoma
  • Unspecified Childhood Solid Tumor, Protocol Specific
Intervention  ICMJE
  • Biological: anti-thymocyte globulin
    Given IV
  • Drug: busulfan
    Given orally
  • Drug: cyclophosphamide
    Given IV
  • Drug: fludarabine phosphate
    Given IV
  • Drug: melphalan
    Given IV
  • Drug: methylprednisolone
    Given IV
  • Radiation: radiation therapy
    Patients undergo radiation therapy two times daily on days -7 to -4.
Study Arms  ICMJE
  • Experimental: Regimen A
    Patients undergo total body irradiation (TBI) two times daily on days -7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and -2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1.
    Interventions:
    • Biological: anti-thymocyte globulin
    • Drug: cyclophosphamide
    • Radiation: radiation therapy
  • Experimental: Regimen B (patients who do not receive TBI)
    Patients receive oral busulfan 4 times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV over 15-20 minutes on days -4 to -2.
    Interventions:
    • Biological: anti-thymocyte globulin
    • Drug: busulfan
    • Drug: melphalan
  • Experimental: Regimen C (patients with Fanconi's anemia/related disorders)
    Patients undergo TBI on day -6. Patients receive ATG IV over at least 6 hours and methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
    Interventions:
    • Biological: anti-thymocyte globulin
    • Drug: cyclophosphamide
    • Drug: fludarabine phosphate
    • Drug: methylprednisolone
    • Radiation: radiation therapy
  • Experimental: Regimen D
    Patients receive oral or IV busulfan 4 times daily on days -9 to -5, ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60 minutes on days -5 to -2.
    Interventions:
    • Biological: anti-thymocyte globulin
    • Drug: busulfan
    • Drug: cyclophosphamide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: November 8, 2006)
25
Original Enrollment  ICMJE Not Provided
Study Completion Date  ICMJE Not Provided
Estimated Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

DISEASE CHARACTERISTICS:

  • Diagnosis of malignant or non-malignant disease, including but not limited to any of the following:

    • Acute myeloid leukemia or acute lymphoblastic leukemia (ALL) with resistant disease beyond first clinical remission (CR)
    • ALL in first CR at high-risk because of 1 of the following factors:

      • Hypoploidy
      • Pseudodiploidy with translocations t(9;22), t(4;11), or t(8;14)
      • Elevated WBC at diagnosis as follows:

        • > 100,000/mm^3 for patients 6-12 months of age
        • > 50,000/mm^3 for patients 10-20 years of age
        • > 20,000/mm^3 for patients 21 years of age
      • Burkitt's lymphoma/leukemia
    • Chronic myelogenous leukemia in first chronic phase or beyond
    • Juvenile myelomonocytic leukemia
    • Advanced stage or relapsed lymphoma
    • Advanced stage or relapsed solid tumors, including any of the following:

      • Neuroblastoma
      • Ewing's sarcoma
      • Rhabdomyosarcoma
    • Myelodysplastic syndromes, excluding patients with grade 3 or 4 myelofibrosis
    • Familial erythrophagocytic histiocytosis
    • Histiocytosis unresponsive to medical management
    • Inborn errors of metabolism
    • Langerhans cell histiocytosis unresponsive to medical management
    • Immune deficiencies, including:

      • Severe combined immune deficiency
      • Wiskott-Aldrich
    • Hemoglobinopathies, including sickle cell disease and thalassemia
    • Severe aplastic anemia
    • Fanconi's anemia
    • Metabolic storage diseases
  • Unrelated cord blood donor must be HLA-identical OR may be mismatched for 1, 2, or 3 HLA-loci (A, B, DR)
  • No other existing HLA-identical related donor available at the time of transplantation

PATIENT CHARACTERISTICS:

Age

  • 21 and under

Performance status

  • Not specified

Life expectancy

  • Not specified

Hematopoietic

  • See Disease Characteristics

Hepatic

  • Not specified

Renal

  • Not specified

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • Not specified

Endocrine therapy

  • Not specified

Radiotherapy

  • Not specified

Surgery

  • Not specified
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00084695
Other Study ID Numbers  ICMJE CDR0000365544
PSCI-2003-232
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Kenneth Gerald Lucas, Penn State Children's Hospital
Study Sponsor  ICMJE Milton S. Hershey Medical Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Kenneth G. Lucas, MD Milton S. Hershey Medical Center
PRS Account National Cancer Institute (NCI)
Verification Date October 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP