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Donor Umbilical Cord Blood Transplantation in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00055653
Recruitment Status : Completed
First Posted : March 7, 2003
Last Update Posted : March 7, 2011
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by:
Roswell Park Cancer Institute

Brief Summary:

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

PURPOSE: Phase II trial to study the effectiveness of allogeneic umbilical cord blood transplantation in treating patients who have leukemia, lymphoma, or nonmalignant hematologic disorders.

Condition or disease Intervention/treatment Phase
Leukemia Lymphoma Myelodysplastic Syndromes Myelodysplastic-Myeloproliferative Diseases Biological: anti-thymocyte globulin Biological: filgrastim Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: fludarabine phosphate Drug: melphalan Drug: methylprednisolone Procedure: umbilical cord blood transplantation Radiation: radiation therapy Phase 2

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Study Type : Interventional  (Clinical Trial)
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Unrelated Umbilical Cord Blood As An Alternate Source Of Stem Cells Transplantation
Study Start Date : January 2003
Actual Primary Completion Date : September 2003
Actual Study Completion Date : September 2005

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of 1 of the following hematologic malignancies:

    • Acute myeloid leukemia (AML)*

      • With or without history of myelodysplastic syndromes (MDS)
      • Patients in first complete remission (CR) (no greater than 5% blasts in marrow) with translocations t(8;21) and inv(16) are allowed provided they failed first-line induction therapy
      • Patients in first CR (no greater than 5% blasts in marrow) with translocations t(15;17) are allowed provided at least 1 of the following is true:

        • Failed first-line induction therapy
        • Molecular evidence of persistent disease
      • No patients in first CR and with Down syndrome
    • Acute lymphoblastic leukemia (ALL)*, meeting 1 of the following criteria:

      • Not in first CR (no greater than 5% blasts in marrow)
      • In first CR and high risk as defined by 1 of the following:

        • Hypoploidy (no more than 44 chromosomes)
        • Pseudodiploidy with translocations or molecular evidence of t(9;22), 11q23, or t(8;14) (excluding B-ALL) or +MLL gene rearrangement
        • One of the following elevated WBC levels:

          • WBC greater than 100,000/mm^3 if 6 to 12 months of age
          • WBC greater than 200,000/mm^3 if between 10 and 17 years of age
          • WBC greater than 20,000/mm^3 if 18 years of age and over (adult [over 18 years of age] patient stratum closed to accrual)
        • Failed to achieve CR after 4 weeks of induction therapy
      • B-ALL that is not in first CR or that meets at least 1 of the high-risk criteria specified above

        • No translocation t(8;14)
        • No blasts with surface immunoglobulins
        • CD10 negative
    • Undifferentiated leukemia*
    • Infant leukemia*
    • Biphenotypic leukemia*
    • Chronic myelogenous leukemia, meeting 1 of the following criteria:

      • Accelerated phase
      • Chronic phase

        • At least 1 year from diagnosis without an identified matched unrelated bone marrow donor AND unresponsive to or unable to tolerate interferon
      • Blast crisis* (greater than 30% promyelocytes plus blasts in the marrow)
    • One of the following MDS:

      • Refractory anemia
      • Refractory anemia with ringed sideroblasts
      • Refractory anemia with excess blasts (RAEB)
      • RAEB in transformation
      • Chronic myelomonocytic leukemia
    • Paroxysmal nocturnal hemoglobinuria
    • Hodgkin's or non-Hodgkin's lymphoma beyond first CR or failed primary induction therapy

      • Tumor displays chemosensitivity (greater than 50% reduction in mass size after the most recent therapy) NOTE: *Patients in third or greater medullary relapse or refractory disease (other than primary induction failures) or blast crisis receive the study busulfan/melphalan conditioning regimen)


  • Diagnosis of one of the following nonmalignant diseases :

    • Acquired severe aplastic anemia (stratum closed to accrual)

      • Unresponsive to medical therapy with anti-thymocyte globulin and/or cyclosporine
    • Inborn errors of metabolism, including, but not limited to the following:

      • Hurler's syndrome
      • Adrenoleukodystrophy
      • Maroteaux-Lamy syndrome
      • Globoid cell leukodystrophy
      • Metachromatic leukodystrophy
      • Fucosidosis
      • Mannosidosis
    • Fanconi anemia documented by increased chromosomal fragility assays and meeting 1 of the following criteria (stratum closed to accrual):

      • Severe pancytopenia

        • Absolute neutrophil count less than 500/mm^3
        • Platelet count less than 20,000/mm^3
        • Hemoglobin less than 8 g/dL
      • Morphologic evidence of MDS with clonal chromosomal abnormalities
      • Leukemia transformation
    • Other marrow failure syndromes, including any of the following (stratum closed to accrual):

      • Blackfan-Diamond syndrome that is unresponsive to medical therapy
      • Kostmann's congenital agranulocytosis unresponsive to medical therapy
      • Congenital amegakaryocytic thrombocytopenia
      • Thrombocytopenia absent radius
    • Combined immune deficiencies including, but not limited to the following:

      • Severe combined immunodeficiency (SCID)
      • Wiskott-Aldrich syndrome
      • Leukocyte adhesion defect
      • Chediak-Higashi disease
      • X-linked lymphoproliferative disease
      • Adenosine deaminase deficiency
      • Purine nucleoside phosphorylase deficiency
      • X-linked SCID
      • Common variable immune deficiency
      • Nezeloff's syndrome
      • Cartilage hair hypoplasia
      • Reticular dysgenesis
  • No active CNS leukemia (cerebrospinal fluid with WBC greater than 5/mm^3 and malignant cells on cytospin)
  • No SCID patients who do not require cytoreduction
  • No dyskeratosis congenita
  • No primary myelofibrosis
  • No grade 3 or greater myelofibrosis
  • Familial erythrophagocytic lymphohistiocytosis patients must not have any of the following:

    • Abnormal brain MRI
    • Neurologic symptoms
    • Lymphocytes and monocytes greater than 7/mm^3 in the cerebrospinal fluid
  • No available 5/6 or 6/6 HLA-matched related donor



  • 55 and under (over 18 closed to accrual)

Performance status

  • Karnofsky 70-100% OR
  • Lansky 50-100% (patients under 16 years old)

Life expectancy

  • Not specified


  • See Disease Characteristics


  • SGOT less than 5 times upper limit of normal
  • Bilirubin less than 2.5 mg/dL


  • Creatinine normal for age OR
  • Creatinine clearance or glomerular filtration rate greater than 50% of lower limit of normal


  • LVEF greater than 40% at rest and must improve with exercise* OR
  • Shortening fraction greater than 26%* NOTE: *If symptomatic


  • DLCO greater than 45% of predicted* (corrected for hemoglobin)
  • FEV_1 and FEC greater than 45% of predicted (corrected for hemoglobin) OR
  • Room air oxygen saturation greater than 85%* NOTE: *If symptomatic


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No uncontrolled viral, bacterial, or fungal infection
  • HIV negative


Biologic therapy

  • See Disease Characteristics
  • More than 12 months since prior allogeneic stem cell transplantation with cytoreductive preparative therapy
  • More than 6 months since prior autologous stem cell transplantation


  • See Biologic therapy

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified


  • No prior enrollment on this study
  • No continuous life support (e.g., mechanical ventilation) within 1 year after study transplantation (for patients with inborn errors of metabolism)

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 identifier (NCT number): NCT00055653

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Sponsors and Collaborators
Roswell Park Cancer Institute
National Heart, Lung, and Blood Institute (NHLBI)
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Study Chair: Philip L. McCarthy, MD Roswell Park Cancer Institute

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Responsible Party: Philip McCarthy, Roswell Park Cancer Institute Identifier: NCT00055653    
Other Study ID Numbers: CDR0000270487
First Posted: March 7, 2003    Key Record Dates
Last Update Posted: March 7, 2011
Last Verified: March 2011
Keywords provided by Roswell Park Cancer Institute:
refractory anemia with excess blasts in transformation
refractory anemia with excess blasts
refractory anemia with ringed sideroblasts
refractory anemia
chronic myelomonocytic leukemia
childhood acute myeloid leukemia in remission
recurrent childhood acute myeloid leukemia
secondary acute myeloid leukemia
childhood acute lymphoblastic leukemia in remission
recurrent childhood acute lymphoblastic leukemia
accelerated phase chronic myelogenous leukemia
blastic phase chronic myelogenous leukemia
chronic phase chronic myelogenous leukemia
acute undifferentiated leukemia
recurrent/refractory childhood Hodgkin lymphoma
recurrent childhood large cell lymphoma
recurrent childhood lymphoblastic lymphoma
recurrent childhood small noncleaved cell lymphoma
untreated childhood acute lymphoblastic leukemia
untreated childhood acute myeloid leukemia and other myeloid malignancies
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
atypical chronic myeloid leukemia
myelodysplastic/myeloproliferative disease, unclassifiable
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Fludarabine phosphate
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs