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

NCT00084695 | Unknown Phase 2

• 2 other identifiers: CDR0000365544PSCI-2003-232
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

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.

Conditions

Childhood Langerhans Cell Histiocytosis; Fanconi Anemia; Leukemia; Lymphoma; Myelodysplastic Syndromes; Neuroblastoma; Sarcoma; Unspecified Childhood Solid Tumor, Protocol Specific

Keywords

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; chronic phase chronic myelogenous leukemia; relapsing chronic myelogenous leukemia; childhood Langerhans cell histiocytosis; recurrent childhood acute myeloid leukemia; recurrent/refractory childhood Hodgkin lymphoma

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (4)

Regimen A

EXPERIMENTAL

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; Drug: cyclophosphamide; Radiation: radiation therapy

Regimen B (patients who do not receive TBI)

EXPERIMENTAL

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; Drug: busulfan; Drug: melphalan

Regimen C (patients with Fanconi's anemia/related disorders)

EXPERIMENTAL

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; Drug: cyclophosphamide; Drug: fludarabine phosphate; Drug: methylprednisolone; Radiation: radiation therapy

Regimen D

EXPERIMENTAL

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; Drug: busulfan; Drug: cyclophosphamide

Interventions

anti-thymocyte globulin BIOLOGICAL

Given IV

Regimen A; Regimen B (patients who do not receive TBI); Regimen C (patients with Fanconi's anemia/related disorders); Regimen D

busulfan DRUG

Given orally

Regimen B (patients who do not receive TBI); Regimen D

cyclophosphamide DRUG

Given IV

Regimen A; Regimen C (patients with Fanconi's anemia/related disorders); Regimen D

fludarabine phosphate DRUG

Given IV

Regimen C (patients with Fanconi's anemia/related disorders)

melphalan DRUG

Given IV

Regimen B (patients who do not receive TBI)

methylprednisolone DRUG

Given IV

Regimen C (patients with Fanconi's anemia/related disorders)

radiation therapy RADIATION

Patients undergo radiation therapy two times daily on days -7 to -4.

Regimen A; Regimen C (patients with Fanconi's anemia/related disorders)

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

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

Sponsors & Collaborators

• Milton S. Hershey Medical Center: lead

Study Sites (1)

Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033-0850, United States

RECRUITING

MeSH Terms

Conditions

Fanconi Anemia; Leukemia; Lymphoma; Myelodysplastic Syndromes; Neuroblastoma; Sarcoma; Neuroectodermal Tumors, Primitive, Peripheral; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myelomonocytic, Juvenile; Burkitt Lymphoma; Dendritic Cell Sarcoma, Interdigitating; Leukemia, Myeloid, Chronic-Phase; Recurrence

Interventions

Antilymphocyte Serum; Busulfan; Cyclophosphamide; fludarabine phosphate; Melphalan; Methylprednisolone; Radiotherapy

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Neoplasms, Connective and Soft Tissue; Leukemia, Lymphoid; Leukemia, Myeloid; Myelodysplastic-Myeloproliferative Diseases; 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; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Myeloproliferative Disorders; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; 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; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Therapeutics

Study Officials

• Kenneth G. Lucas, MD

  Milton S. Hershey Medical Center

  STUDY CHAIR

Study Design

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

Study Record Dates

• First Submitted: June 10, 2004
• First Posted: June 11, 2004
• Study Start: September 1, 2003
• Primary Completion: December 1, 2012
• Last Updated: January 10, 2014

Record last verified: 2008-10

Locations

US (1)