Clinical trial NCT00084695: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: 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
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2004-06-11
Enrollment: 25
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Impact of the use of umbilical cord blood as a source of hematopoietic stem cells
measure: Comparison of the incidence of graft-vs-host disease with historical data
measure: Comparison of the incidence of engraftment with historical data
Secondary outcomes:
Other outcomes:
Locations:
city: Hershey
country: United States
facility: Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
state: Pennsylvania
Study officials:
affiliation: Milton S. Hershey Medical Center
name: Kenneth G. Lucas, MD
role: STUDY_CHAIR
References: