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

January 9, 2014 updated by: Milton S. Hershey Medical Center

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

25

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033-0850
        • Recruiting
        • Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
        • Contact:
          • Kenneth G. Lucas, MD
          • Phone Number: 717-531-6012
          • Email: klucas@psu.edu

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Drug: cyclophosphamide
Given IV
Biological: anti-thymocyte globulin
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.
Drug: busulfan
Given orally
Drug: melphalan
Given IV
Biological: anti-thymocyte globulin
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.
Drug: fludarabine phosphate
Given IV
Drug: cyclophosphamide
Given IV
Drug: methylprednisolone
Given IV
Biological: anti-thymocyte globulin
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.
Drug: busulfan
Given orally
Drug: cyclophosphamide
Given IV
Biological: anti-thymocyte globulin
Given IV

What is the study measuring?

Primary Outcome Measures

Outcome Measure
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Milton S. Hershey Medical Center

Investigators

  • Study Chair: Kenneth G. Lucas, MD, Milton S. Hershey Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2003

Primary Completion (Anticipated)

December 1, 2012

Study Registration Dates

First Submitted

June 10, 2004

First Submitted That Met QC Criteria

June 10, 2004

First Posted (Estimate)

June 11, 2004

Study Record Updates

Last Update Posted (Estimate)

January 10, 2014

Last Update Submitted That Met QC Criteria

January 9, 2014

Last Verified

October 1, 2008

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CDR0000365544
  • PSCI-2003-232

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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