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

Lymphoma Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00084695
• Other study ID #: CDR0000365544
• Secondary ID: PSCI-2003-232
• Status: Recruiting
• Phase: Phase 2
• First received: June 10, 2004
• Last updated: January 9, 2014
• Start date: September 2003

Study information

• Verified date: October 2008
• Source: National Cancer Institute (NCI)
• Contact: n/a
• Is FDA regulated: No
• Health authority: Unspecified
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 21 Years

Eligibility

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

Study Design

Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Childhood Langerhans Cell Histiocytosis
• Fanconi Anemia
• Fanconi Syndrome
• Histiocytosis
• Histiocytosis, Langerhans-Cell
• Leukemia
• Lymphoma
• Myelodysplastic Syndromes
• Neuroblastoma
• Preleukemia
• Sarcoma
• Unspecified Childhood Solid Tumor, Protocol Specific

Intervention

Biological:
• anti-thymocyte globulin
Given IV

Drug:
• busulfan
Given orally
• cyclophosphamide
Given IV
• fludarabine phosphate
Given IV
• melphalan
Given IV
• methylprednisolone
Given IV

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

Locations

Country	Name	City	State
United States	Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center	Hershey	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Milton S. Hershey Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Impact of the use of umbilical cord blood as a source of hematopoietic stem cells			No
Primary	Comparison of the incidence of graft-vs-host disease with historical data			No
Primary	Comparison of the incidence of engraftment with historical data			No