or
forgot password

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


Phase 2
N/A
21 Years
Open (Enrolling)
Both
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific

Thank you

Trial Information

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


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.

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

Type of Study:

Interventional

Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Impact of the use of umbilical cord blood as a source of hematopoietic stem cells

Safety Issue:

No

Principal Investigator

Kenneth G. Lucas, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Milton S. Hershey Medical Center

Authority:

Unspecified

Study ID:

CDR0000365544

NCT ID:

NCT00084695

Start Date:

September 2003

Completion Date:

Related Keywords:

  • Childhood Langerhans Cell Histiocytosis
  • Fanconi Anemia
  • Leukemia
  • Lymphoma
  • Myelodysplastic Syndromes
  • Neuroblastoma
  • Sarcoma
  • Unspecified Childhood Solid Tumor, Protocol Specific
  • 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
  • Anemia
  • Fanconi Anemia
  • Fanconi Syndrome
  • Histiocytosis
  • Histiocytosis, Langerhans-Cell
  • Leukemia
  • Lymphoma
  • Myelodysplastic Syndromes
  • Preleukemia
  • Neuroblastoma
  • Lymphoma, Non-Hodgkin
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma

Name

Location
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center Hershey, Pennsylvania  17033-0850