Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Nonmalignant Hematologic Disease

NCT00003913 | Completed Phase 2

• 5 other identifiers: 1330.00FHCRC-1330.00UMN-MT-9817NCI-G99-1523CDR0000067092
• Study Type: interventional
• Target: 390
• Locations: 1 country
• Sites: 23

Brief Summary

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. 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 umbilical cord blood transplantation plus combination chemotherapy in treating patients who have hematologic cancer or nonmalignant hematologic disease.

Conditions

Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases

Keywords

recurrent childhood acute lymphoblastic leukemia; recurrent childhood lymphoblastic lymphoma; recurrent childhood acute myeloid leukemia; relapsing chronic myelogenous leukemia; chronic phase chronic myelogenous leukemia; accelerated phase chronic myelogenous leukemia; childhood acute myeloid leukemia in remission; childhood acute lymphoblastic leukemia in remission; recurrent/refractory childhood Hodgkin lymphoma; refractory anemia; refractory anemia with ringed sideroblasts; refractory anemia with excess blasts; refractory anemia with excess blasts in transformation; chronic myelomonocytic leukemia; acute undifferentiated leukemia; secondary acute myeloid leukemia; de novo myelodysplastic syndromes; previously treated myelodysplastic syndromes; secondary myelodysplastic syndromes; recurrent childhood small noncleaved cell lymphoma; recurrent childhood large cell lymphoma; juvenile myelomonocytic leukemia; blastic phase chronic myelogenous leukemia; childhood chronic myelogenous leukemia; atypical chronic myeloid leukemia; myelodysplastic/myeloproliferative disease, unclassifiable

Interventions

anti-thymocyte globulin BIOLOGICAL

filgrastim BIOLOGICAL

busulfan DRUG

cyclophosphamide DRUG

methylprednisolone DRUG

umbilical cord blood transplantation PROCEDURE

radiation therapy RADIATION

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

DISEASE CHARACTERISTICS: * One of the following diagnoses: * Acute myeloid leukemia (AML), with or without myelodysplastic syndromes * Not in first complete remission (CR)\* with translocations t(8;21) and inv (16) unless failure of first-line induction therapy * Not in first CR\* with translocations t(15;17) abnormality unless: * Failure of first-line induction therapy OR * Molecular evidence of persistent disease * Not in first CR with Down syndrome * Patients with third or greater medullary relapse or refractory disease (other than primary induction failures) receive busulfan/melphalan conditioning regimen NOTE: \* CR defined by no greater than 5% blasts in marrow * Acute lymphocytic leukemia (ALL) * Not in first CR OR * High-risk ALL in first CR, with high risk defined as one of the following: * Hypoploidy (no greater than 44 chromosomes) * Pseudodiploidy with translocations or molecular evidence of t(9;22), 11q23, or t(8;14) (except B-cell ALL) with or without MLL gene arrangement * Elevated WBC at presentation * Age 6-12 months: greater than 100,000/mm\^3 * Age 10-17 years: greater than 200,000/mm\^3 * Age 18: greater than 20,000/mm\^3 * Failed to achieve CR after 4 weeks of induction therapy * Patients with B-ALL must not be in first CR, must meet at least one of the high-risk criteria specified above, or must not meet any of the following criteria: * Translocation t(8;14) * Blasts have surface immunoglobulins * CD10 positive * Patients with third or greater medullary relapse or refractory disease (other than primary induction failures) receive busulfan/melphalan conditioning regimen * Chronic myelogenous leukemia, meeting criteria for 1 of the following: * Accelerated phase * Chronic phase if 1 year from diagnosis without a matched unrelated bone marrow donor AND unresponsive to or unable to tolerate interferon * Blast crisis, defined as greater than 30% promyelocytes plus blasts in bone marrow * Patients receive busulfan/melphalan conditioning regimen * Acute undifferentiated leukemia (AUL), infant leukemia, or biphenotypic leukemia * Patients with third or greater medullary relapse or refractory disease (other than primary induction failures) receive busulfan/melphalan conditioning regimen * Juvenile myelomonocytic leukemia meeting the following criteria: * No Philadelphia chromosome * Bone marrow blasts less than 30% * Peripheral blood monocytes greater than 1,000/mm\^3 * At least 2 of the following: * Peripheral blood spontaneous growth and/or sargramostim (GM-CSF) hypersensitivity * Increased hemoglobin F for age * Clonal abnormalities (e.g., monosomy 7 or RAS mutations) * Peripheral blood with myeloid precursors * WBC greater than 10,000/mm\^3 * Myelodysplastic syndromes defined by the following: * Refractory anemia (RA) * RA with ringed sideroblasts * RA with excess blasts (RAEB) * RAEB in transformation * Chronic myelomonocytic leukemia * Paroxysmal nocturnal hemoglobinuria * Hodgkin's lymphoma or non-Hodgkin's lymphoma beyond first CR or primary induction failures AND chemosensitive (greater than 50% reduction in tumor mass size) * Inborn error of metabolism including, but not limited to, Hurler's syndrome, adrenoleukodystrophy (ALD), Maroteaux-Lamy syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy, fucosidosis, or mannosidosis * For ALD patients over age 5, IQ must be at least 80 * For all other patients over age 5, IQ must be at least 70 * For all patients age 5 and under, developmental quotient or clinical neurodevelopmental examination should demonstrate potential for stabilization at a level of functioning where continuous life support (e.g., mechanical ventilation) would not be predicted to be required in the year after transplantation * Combined immune deficiencies including, but not limited to: * Severe combined immunodeficiency (SCID) requiring cytoreduction * 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 * Nezelof's syndrome * Cartilage hair hypoplasia * No dyskeratosis congenita * No ALL, AML, AUL, or biphenotypic leukemia in third or higher medullary relapse or refractory disease other than primary induction failure * No primary myelofibrosis or myelofibrosis grade 3 or worse * No active CNS leukemia involvement (CSF with WBC greater than 5/mm\^3 and malignant cells on cytospin) * No consenting 5/6 or 6/6 HLA-matched related donor available * 3-6/6 HLA-matched unrelated umbilical cord blood donor available PATIENT CHARACTERISTICS: Age: * See Disease Characteristics * 18 and under Performance status: * Karnofsky 70-100%, if age 16 to 18 * Lansky 50-100%, if under age 16 Life expectancy: * Not specified Hematopoietic: * See Disease Characteristics Hepatic: * Bilirubin less than 2.5 mg/dL * SGOT less than 5 times upper limit of normal Renal: * Creatinine normal for age OR * Creatinine clearance or glomerular filtration rate greater than 50% lower limit of normal for age Cardiovascular: * If symptomatic: * LVEF greater than 40% (or shortening fraction greater than 26%) and improves with exercise OR * Shortening fraction greater than 26% Pulmonary: * If symptomatic: * DLCO, FEV\_1, and FEC greater than 45% predicted OR * Oxygen saturation greater than 85% on room air Other: * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * HIV negative * No uncontrolled viral, bacterial, or fungal infection PRIOR CONCURRENT THERAPY: Biologic therapy: * See Disease Characteristics * At least 1 year since prior allogeneic stem cell transplantation (SCT) with cytoreductive preparative therapy * At least 6 months since prior autologous SCT * No concurrent thrombopoietic growth factors Chemotherapy: * See Disease Characteristics * See Biologic therapy Endocrine therapy: * Not specified Radiotherapy: * Not specified Surgery: * Not specified

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Fred Hutchinson Cancer Center: lead
• National Cancer Institute (NCI): collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (23)

City of Hope Comprehensive Cancer Center

Duarte, California, 91010-3000, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027-0700, United States

Location

Jonsson Comprehensive Cancer Center, UCLA

Los Angeles, California, 90095-1781, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010-2970, United States

Location

Indiana University Cancer Center

Indianapolis, Indiana, 46202-5289, United States

Location

Children's Hospital of New Orleans

New Orleans, Louisiana, 70118, United States

Location

Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support

Bethesda, Maryland, 20892-1182, United States

Location

Warren Grant Magnuson Clinical Center

Bethesda, Maryland, 20892-1182, United States

Location

Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Spectrum Health and DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

University of Minnesota Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States

Location

Cancer Center at Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

North Shore University Hospital

Manhasset, New York, 11030, United States

Location

James P. Wilmot Cancer Center at University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Duke Comprehensive Cancer Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Ireland Cancer Center

Cleveland, Ohio, 44106-5065, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Medical City Dallas Hospital

Dallas, Texas, 75230, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic-Myeloproliferative Diseases; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Chronic-Phase; Leukemia, Myeloid, Accelerated Phase; Recurrence; Anemia, Refractory; Anemia, Refractory, with Excess of Blasts; Leukemia, Myelomonocytic, Chronic; Leukemia, Biphenotypic, Acute; Burkitt Lymphoma; Dendritic Cell Sarcoma, Interdigitating; Leukemia, Myelomonocytic, Juvenile; Blast Crisis; Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative; Myeloproliferative Disorders

Interventions

Antilymphocyte Serum; Filgrastim; Busulfan; Cyclophosphamide; Methylprednisolone; Cord Blood Stem Cell Transplantation; Radiotherapy

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Bone Marrow Diseases; Leukemia, Lymphoid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Anemia; Epstein-Barr Virus Infections; Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Histiocytic Disorders, Malignant; Histiocytosis; Cell Transformation, Neoplastic; Carcinogenesis; Neoplastic Processes

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Biological Factors; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Colleen Delaney, MD, MSC

  Fred Hutchinson Cancer Center

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: November 1, 1999
• First Posted: January 27, 2003
• Study Start: December 1, 1998
• Primary Completion: August 1, 2005
• Study Completion: August 1, 2005
• Last Updated: April 2, 2010

Record last verified: 2010-03

Locations

US (23)