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

NCT ID: NCT00084695

Last Updated: 2014-01-10

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2003-09-30

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.

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 0.
• 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.

Conditions

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

Study Design

• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

anti-thymocyte globulin

Intervention Type: BIOLOGICAL

Given IV

cyclophosphamide

Intervention Type: DRUG

Given IV

radiation therapy

Intervention Type: RADIATION

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

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.

Group Type: EXPERIMENTAL

anti-thymocyte globulin

Intervention Type: BIOLOGICAL

Given IV

busulfan

Intervention Type: DRUG

Given orally

melphalan

Intervention Type: DRUG

Given IV

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.

Group Type: EXPERIMENTAL

anti-thymocyte globulin

Intervention Type: BIOLOGICAL

Given IV

cyclophosphamide

Intervention Type: DRUG

Given IV

fludarabine phosphate

Intervention Type: DRUG

Given IV

methylprednisolone

Intervention Type: DRUG

Given IV

radiation therapy

Intervention Type: RADIATION

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

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.

Group Type: EXPERIMENTAL

anti-thymocyte globulin

Intervention Type: BIOLOGICAL

Given IV

busulfan

Intervention Type: DRUG

Given orally

cyclophosphamide

Intervention Type: DRUG

Given IV

Interventions

anti-thymocyte globulin

Given IV

Intervention Type: BIOLOGICAL

busulfan

Given orally

Intervention Type: DRUG

cyclophosphamide

Given IV

Intervention Type: DRUG

fludarabine phosphate

Given IV

Intervention Type: DRUG

melphalan

Given IV

Intervention Type: DRUG

methylprednisolone

Given IV

Intervention Type: DRUG

radiation therapy

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

Intervention Type: RADIATION

Eligibility Criteria

Inclusion Criteria

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

• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Milton S. Hershey Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Penn State Children's Hospital

Principal Investigators

Kenneth G. Lucas, MD

Role: STUDY_CHAIR

Milton S. Hershey Medical Center

Locations

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

Hershey, Pennsylvania, United States

Site Status: RECRUITING

Countries

United States

Facility Contacts

Kenneth G. Lucas, MD

Role: primary

klucas@psu.edu

717-531-6012

Other Identifiers

PSCI-2003-232

Identifier Type: -

Identifier Source: secondary_id

CDR0000365544

Identifier Type: -

Identifier Source: org_study_id