Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
The recruitment status of this study is unknown because the information has not been verified recently.
Verified October 2008 by National Cancer Institute (NCI).
Recruitment status was Recruiting
Recruitment status was Recruiting
Sponsor:
Milton S. Hershey Medical Center
Information provided by:
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00084695
First received: June 10, 2004
Last updated: March 13, 2012
Last verified: October 2008
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Purpose
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 | Intervention | 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 |
| Study Type: | Interventional |
| Study Design: | Masking: Open Label Primary Purpose: Treatment |
| Official Title: | The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells |
Resource links provided by NLM:
Genetics Home Reference related topics:
Ewing sarcoma
Fanconi anemia
Langerhans cell histiocytosis
neuroblastoma
MedlinePlus related topics:
Anemia
Cancer
Hodgkin Disease
Leukemia
Lymphoma
Myelodysplastic Syndromes
Neuroblastoma
Soft Tissue Sarcoma
Drug Information available for:
Cyclophosphamide
Prednisolone
Prednisolone acetate
Methylprednisolone acetate
Busulfan
Methylprednisolone
Prednisolone sodium phosphate
Melphalan
Prednisolone phosphate
Prednisolone sodium succinate
Methylprednisolone sodium succinate
Melphalan hydrochloride
Fludarabine
Fludarabine phosphate
Antilymphocyte Serum
U.S. FDA Resources
Further study details as provided by National Cancer Institute (NCI):
Primary Outcome Measures:
- Impact of the use of umbilical cord blood as a source of hematopoietic stem cells [ Designated as safety issue: No ]
- Comparison of the incidence of graft-vs-host disease with historical data [ Designated as safety issue: No ]
- Comparison of the incidence of engraftment with historical data [ Designated as safety issue: No ]
| Estimated Enrollment: | 25 |
| Study Start Date: | September 2003 |
| Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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
|
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.
Eligibility| Ages Eligible for Study: | up to 21 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00084695
Locations
| United States, Pennsylvania | |
| Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center | Recruiting |
| Hershey, Pennsylvania, United States, 17033-0850 | |
| Contact: Kenneth G. Lucas, MD 717-531-6012 klucas@psu.edu | |
Sponsors and Collaborators
Milton S. Hershey Medical Center
Investigators
| Study Chair: | Kenneth G. Lucas, MD | Milton S. Hershey Medical Center |
More Information
Additional Information:
No publications provided
| Responsible Party: | Kenneth Gerald Lucas, Penn State Children's Hospital |
| ClinicalTrials.gov Identifier: | NCT00084695 History of Changes |
| Other Study ID Numbers: | CDR0000365544, PSCI-2003-232 |
| Study First Received: | June 10, 2004 |
| Last Updated: | March 13, 2012 |
| Health Authority: | Unspecified |
Keywords provided by National Cancer Institute (NCI):
|
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 |
Additional relevant MeSH terms:
|
Anemia Fanconi Anemia Fanconi Syndrome Histiocytosis Histiocytosis, Langerhans-Cell Leukemia Lymphoma Myelodysplastic Syndromes Preleukemia Neuroblastoma Lymphoma, Non-Hodgkin Neuroectodermal Tumors, Primitive, Peripheral Sarcoma Hematologic Diseases Anemia, Hypoplastic, Congenital |
Anemia, Aplastic Bone Marrow Diseases Genetic Diseases, Inborn DNA Repair-Deficiency Disorders Metabolic Diseases Kidney Diseases Urologic Diseases Renal Tubular Transport, Inborn Errors Metabolism, Inborn Errors Lymphatic Diseases Lung Diseases, Interstitial Lung Diseases Respiratory Tract Diseases Neoplasms by Histologic Type Neoplasms |
ClinicalTrials.gov processed this record on May 23, 2013