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

Primary Purpose

Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia

Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
anti-thymocyte globulin
busulfan
cyclophosphamide
fludarabine phosphate
melphalan
methylprednisolone
radiation therapy
Sponsored by
Milton S. Hershey Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Childhood Langerhans Cell Histiocytosis focused on measuring 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

Eligibility Criteria

undefined - 21 Years (Child, Adult)All SexesDoes not accept 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 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

Sites / Locations

  • Penn State Hershey Cancer Institute at Milton S. Hershey Medical CenterRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Regimen A

Regimen B (patients who do not receive TBI)

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

Regimen D

Arm Description

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.

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.

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.

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.

Outcomes

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

Secondary Outcome Measures

Full Information

First Posted
June 10, 2004
Last Updated
January 9, 2014
Sponsor
Milton S. Hershey Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00084695
Brief Title
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
Official Title
The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells
Study Type
Interventional

2. Study Status

Record Verification Date
October 2008
Overall Recruitment Status
Unknown status
Study Start Date
September 2003 (undefined)
Primary Completion Date
December 2012 (Anticipated)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Milton S. Hershey Medical Center

4. Oversight

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Masking
None (Open Label)
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Regimen A
Arm Type
Experimental
Arm Description
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.
Arm Title
Regimen B (patients who do not receive TBI)
Arm Type
Experimental
Arm Description
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.
Arm Title
Regimen C (patients with Fanconi's anemia/related disorders)
Arm Type
Experimental
Arm Description
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.
Arm Title
Regimen D
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
anti-thymocyte globulin
Intervention Description
Given IV
Intervention Type
Drug
Intervention Name(s)
busulfan
Intervention Description
Given orally
Intervention Type
Drug
Intervention Name(s)
cyclophosphamide
Intervention Description
Given IV
Intervention Type
Drug
Intervention Name(s)
fludarabine phosphate
Intervention Description
Given IV
Intervention Type
Drug
Intervention Name(s)
melphalan
Intervention Description
Given IV
Intervention Type
Drug
Intervention Name(s)
methylprednisolone
Intervention Description
Given IV
Intervention Type
Radiation
Intervention Name(s)
radiation therapy
Intervention Description
Patients undergo radiation therapy two times daily on days -7 to -4.
Primary Outcome Measure Information:
Title
Impact of the use of umbilical cord blood as a source of hematopoietic stem cells
Title
Comparison of the incidence of graft-vs-host disease with historical data
Title
Comparison of the incidence of engraftment with historical data

10. Eligibility

Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kenneth G. Lucas, MD
Organizational Affiliation
Milton S. Hershey Medical Center
Official's Role
Study Chair
Facility Information:
Facility Name
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033-0850
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kenneth G. Lucas, MD
Phone
717-531-6012
Email
klucas@psu.edu

12. IPD Sharing Statement

Learn more about this trial

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

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