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

Sponsor

Milton S. Hershey Medical Center (Other)

Overall Status

Unknown status

CT.gov ID

NCT00084695

Collaborator

(none)

Enrollment

Location

Arms

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	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

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

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

25 participants

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells

Study Start Date :

Sep 1, 2003

Anticipated Primary Completion Date :

Dec 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

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 []

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 21 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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