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

This study has been completed.
Sponsor:
Collaborator:
Information provided by:
Roswell Park Cancer Institute
ClinicalTrials.gov Identifier:
NCT00055653
First received: March 6, 2003
Last updated: March 3, 2011
Last verified: March 2011
  Purpose

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 Intervention 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

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: Unrelated Umbilical Cord Blood As An Alternate Source Of Stem Cells Transplantation

Resource links provided by NLM:


Further study details as provided by Roswell Park Cancer Institute:

Study Start Date: January 2003
Study Completion Date: September 2005
Primary Completion Date: September 2003 (Final data collection date for primary outcome measure)
  Show Detailed Description

  Eligibility

Ages Eligible for Study:   up to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • 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)

OR

  • 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

PATIENT CHARACTERISTICS:

Age

  • 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

Hematopoietic

  • See Disease Characteristics

Hepatic

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

Renal

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

Cardiovascular

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

Pulmonary

  • 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

Other

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

PRIOR CONCURRENT THERAPY:

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

Chemotherapy

  • See Biologic therapy

Endocrine therapy

  • Not specified

Radiotherapy

  • Not specified

Surgery

  • Not specified

Other

  • 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)
  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: NCT00055653

  Hide Study Locations
Locations
United States, California
City of Hope Comprehensive Cancer Center
Duarte, California, United States, 91010-3000
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027-0700
Jonsson Comprehensive Cancer Center, UCLA
Los Angeles, California, United States, 90095-1781
Children's Hospital of Orange County
Orange, California, United States, 92868
Children's Medical Center, University of California San Francisco
San Francisco, California, United States, 94143-1278
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Indiana
Indiana University Cancer Center
Indianapolis, Indiana, United States, 46202-5289
United States, Louisiana
Children's Hospital of New Orleans
New Orleans, Louisiana, United States, 70118
United States, Massachusetts
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
United States, Michigan
Spectrum Health and DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
United States, Minnesota
University of Minnesota Cancer Center
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Cardinal Glennon Children's Hospital
Saint Louis, Missouri, United States, 63104-1095
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, New York
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263-0001
North Shore University Hospital
Manhasset, New York, United States, 11030
James P. Wilmot Cancer Center at University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, North Carolina
Duke Comprehensive Cancer Center
Durham, North Carolina, United States, 27710
United States, Ohio
Ireland Cancer Center
Cleveland, Ohio, United States, 44106-5065
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, United States, 37232-6310
United States, Texas
Children's Medical Center of Dallas
Dallas, Texas, United States, 75235
Medical City Dallas Hospital
Dallas, Texas, United States, 75230
Texas Transplant Institute
San Antonio, Texas, United States, 78229
United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109-1024
Sponsors and Collaborators
Roswell Park Cancer Institute
Investigators
Study Chair: Philip L. McCarthy, MD Roswell Park Cancer Institute
  More Information

Additional Information:
No publications provided

Responsible Party: Philip McCarthy, Roswell Park Cancer Institute
ClinicalTrials.gov Identifier: NCT00055653     History of Changes
Other Study ID Numbers: CDR0000270487, RPCI-DS-00-22
Study First Received: March 6, 2003
Last Updated: March 3, 2011
Health Authority: United States: Food and Drug Administration

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:
Hematologic Diseases
Leukemia
Lymphoma
Myelodysplastic Syndromes
Preleukemia
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Precancerous Conditions
Antilymphocyte Serum
Busulfan
Cyclophosphamide
Cyclosporins
Cyclosporine
Melphalan
Fludarabine monophosphate
Lenograstim
Fludarabine
Methylprednisolone Hemisuccinate
Prednisolone
Vidarabine
Methylprednisolone acetate
Prednisolone acetate
Methylprednisolone

ClinicalTrials.gov processed this record on July 20, 2014