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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified October 2008 by National Cancer Institute (NCI).
Recruitment status was  Recruiting
Sponsor:
Information provided by:
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00084695
First received: June 10, 2004
Last updated: January 9, 2014
Last verified: October 2008
  Purpose

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.


Condition Intervention Phase
Childhood Langerhans Cell Histiocytosis
Fanconi Anemia
Leukemia
Lymphoma
Myelodysplastic Syndromes
Neuroblastoma
Sarcoma
Unspecified Childhood Solid Tumor, Protocol Specific
Biological: anti-thymocyte globulin
Drug: busulfan
Drug: cyclophosphamide
Drug: fludarabine phosphate
Drug: melphalan
Drug: methylprednisolone
Radiation: radiation therapy
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Impact of the use of umbilical cord blood as a source of hematopoietic stem cells [ Designated as safety issue: No ]
  • Comparison of the incidence of graft-vs-host disease with historical data [ Designated as safety issue: No ]
  • Comparison of the incidence of engraftment with historical data [ Designated as safety issue: No ]

Estimated Enrollment: 25
Study Start Date: September 2003
Estimated Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Biological: anti-thymocyte globulin
Given IV
Drug: cyclophosphamide
Given IV
Radiation: radiation therapy
Patients undergo radiation therapy two times daily on days -7 to -4.
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.
Biological: anti-thymocyte globulin
Given IV
Drug: busulfan
Given orally
Drug: melphalan
Given IV
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.
Biological: anti-thymocyte globulin
Given IV
Drug: cyclophosphamide
Given IV
Drug: fludarabine phosphate
Given IV
Drug: methylprednisolone
Given IV
Radiation: radiation therapy
Patients undergo radiation therapy two times daily on days -7 to -4.
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.
Biological: anti-thymocyte globulin
Given IV
Drug: busulfan
Given orally
Drug: cyclophosphamide
Given IV

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.

  Eligibility

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

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
  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: NCT00084695

Locations
United States, Pennsylvania
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center Recruiting
Hershey, Pennsylvania, United States, 17033-0850
Contact: Kenneth G. Lucas, MD    717-531-6012    klucas@psu.edu   
Sponsors and Collaborators
Milton S. Hershey Medical Center
Investigators
Study Chair: Kenneth G. Lucas, MD Milton S. Hershey Medical Center
  More Information

Additional Information:
No publications provided

Responsible Party: Kenneth Gerald Lucas, Penn State Children's Hospital
ClinicalTrials.gov Identifier: NCT00084695     History of Changes
Other Study ID Numbers: CDR0000365544, PSCI-2003-232
Study First Received: June 10, 2004
Last Updated: January 9, 2014
Health Authority: Unspecified

Keywords provided by National Cancer Institute (NCI):
childhood myelodysplastic syndromes
recurrent childhood rhabdomyosarcoma
unspecified childhood solid tumor, protocol specific
previously treated childhood rhabdomyosarcoma
previously untreated childhood rhabdomyosarcoma
disseminated neuroblastoma
regional neuroblastoma
recurrent neuroblastoma
metastatic Ewing sarcoma/peripheral primitive neuroectodermal tumor
recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor
recurrent childhood acute lymphoblastic leukemia
juvenile myelomonocytic leukemia
childhood acute lymphoblastic leukemia in remission
childhood Burkitt lymphoma
recurrent childhood lymphoblastic lymphoma
stage III childhood lymphoblastic lymphoma
stage IV childhood lymphoblastic lymphoma
recurrent childhood small noncleaved cell lymphoma
stage III childhood small noncleaved cell lymphoma
stage IV childhood small noncleaved cell lymphoma
recurrent childhood large cell lymphoma
stage III childhood large cell lymphoma
stage IV childhood large cell lymphoma
stage III childhood Hodgkin lymphoma
stage IV childhood Hodgkin lymphoma
previously treated myelodysplastic syndromes
Fanconi anemia
de novo myelodysplastic syndromes
secondary myelodysplastic syndromes
childhood chronic myelogenous leukemia

Additional relevant MeSH terms:
Fanconi Anemia
Fanconi Syndrome
Histiocytosis
Histiocytosis, Langerhans-Cell
Leukemia
Lymphoma
Myelodysplastic Syndromes
Neuroblastoma
Preleukemia
Sarcoma
Syndrome
Anemia
Anemia, Aplastic
Anemia, Hypoplastic, Congenital
Bone Marrow Diseases
Disease
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Immune System Diseases
Immunoproliferative Disorders
Kidney Diseases
Lung Diseases
Lung Diseases, Interstitial
Lymphatic Diseases
Lymphoproliferative Disorders
Metabolic Diseases
Metabolism, Inborn Errors
Neoplasms
Neoplasms by Histologic Type

ClinicalTrials.gov processed this record on October 29, 2014