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Total-Body Irradiation, Thiotepa, and Fludarabine in Treating Young Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer

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ClinicalTrials.gov Identifier: NCT00112567
Recruitment Status : Completed
First Posted : June 3, 2005
Last Update Posted : September 21, 2010
National Cancer Institute (NCI)
Information provided by:
Fred Hutchinson Cancer Research Center

Brief Summary:

RATIONALE: Chemotherapy, such as fludarabine and thiotepa, and radiation therapy may destroy cancerous blood-forming cells (stem cells) in the blood and bone marrow. Giving healthy stem cells from a donor whose blood closely resembles the patient's blood will help the patient's bone marrow make new stem cells that become red blood cells, white blood cells, and platelets.

PURPOSE: This phase I/II trial is studying the side effects of total-body irradiation, fludarabine, and thiotepa and to see how well they work in treating young patients who are undergoing a donor stem cell transplant for hematologic cancer.

Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic Syndromes Drug: fludarabine phosphate Drug: thiotepa Procedure: peripheral blood stem cell transplantation Radiation: radiation therapy Phase 1 Phase 2

Detailed Description:



  • Determine the safety of a conditioning regimen without anti-thymocyte globulin comprising total body irradiation, thiotepa, and fludarabine followed by CD34-positive-selected haploidentical allogeneic peripheral blood stem cell transplantation in young patients with life-threatening hematologic malignancies.


  • Determine the risk for severe graft-vs-host disease in patients treated with this regimen.
  • Determine the kinetics of immune reconstitution in patients treated with this regimen.
  • Determine the risk for life-threatening infections in patients treated with this regimen.


  • Conditioning regimen: Patients 7 years of age and under undergo total body irradiation twice daily on days -9 to -7. Patients over 7 years of age undergo total body irradiation once on day -7. All patients receive fludarabine IV once daily on days -6 to -2 and thiotepa IV over 2 hours twice on day -5.
  • CD34-positive (CD34+)-selected haploidentical allogeneic peripheral blood stem cell transplantation (PBSCT): Patients undergo CD34+-selected allogeneic PBSCT on days 0 and 2.

Patients with acute lymphoblastic leukemia or CNS disease also receive methotrexate intrathecally twice before transplantation and 4 times after day 35 post-transplantation. Male patients with lymphoid malignancies undergo additional radiotherapy to the testes.

After completion of study treatment, patients are followed for at least 100 days, at 1 year, and then periodically thereafter.

PROJECTED ACCRUAL: A total of 20 patients (10 patients ≤ 7 years of age and 10 patients > 7 years of age) will be accrued for this study within 3 years.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Study of Total Body Irradiation, Thiotepa, and Fludarabine as Conditioning for Haploidentical CD34+ Purified Peripheral Blood Stem Cell Transplants
Study Start Date : April 2003
Estimated Study Completion Date : July 2007

Primary Outcome Measures :
  1. Safety

Secondary Outcome Measures :
  1. Risk of severe graft-versus-host disease
  2. Kinetics of immune reconstitution
  3. Risk of life-threatening infections

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of a life-threatening hematologic malignancy, including any of the following:

    • Acute leukemia advanced beyond first remission
    • Acute leukemia in first remission* with very high-risk prognostic features, including any of the following:

      • Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL)
      • ALL or acute myeloid leukemia (AML) with 11q23 chromosomal abnormality
      • Hypodiploid ALL
      • Failed to achieve first remission within 1 month after induction therapy
      • Secondary AML
    • Myelodysplastic syndromes with International Prognostic Index score > 1
    • Chronic myelogenous leukemia in accelerated or blast phase NOTE: *Must be approved by PCC
  • Haploidentical family donor available

    • No suitable HLA-matched related or unrelated donor available
    • No related donor mismatched for a single HLA-A, -B, -C, -DRB1, or -DQB1 antigen available



  • Under 21

Performance status

  • Not specified

Life expectancy

  • At least 6 months


  • Not specified


  • SGPT and SGOT < 2 times upper limit of normal (ULN)*
  • Bilirubin < 2 times ULN* NOTE: *Unless due to malignancy


  • Not specified


  • Ejection fraction ≥ 45%


  • DLCO ≥ 60% of predicted


  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • HIV negative


Biologic therapy

  • No second bone marrow transplantation, after a first regimen containing total body irradiation
  • No concurrent growth factors until day 21 post-transplantation


  • Not specified

Endocrine therapy

  • Not specified


  • See Biologic therapy


  • Not specified

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00112567

United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98104
Seattle Cancer Care Alliance
Seattle, Washington, United States, 98109-1023
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
National Cancer Institute (NCI)
Principal Investigator: Ann E. Woolfrey, MD Fred Hutchinson Cancer Research Center

ClinicalTrials.gov Identifier: NCT00112567     History of Changes
Other Study ID Numbers: 1629.00
CDR0000430650 ( Registry Identifier: PDQ )
First Posted: June 3, 2005    Key Record Dates
Last Update Posted: September 21, 2010
Last Verified: September 2010

Keywords provided by Fred Hutchinson Cancer Research Center:
recurrent childhood acute lymphoblastic leukemia
childhood myelodysplastic syndromes
childhood acute lymphoblastic leukemia in remission
recurrent childhood acute myeloid leukemia
childhood acute myeloid leukemia in remission
secondary acute myeloid leukemia
accelerated phase chronic myelogenous leukemia
blastic phase chronic myelogenous leukemia
childhood chronic myelogenous leukemia
relapsing chronic myelogenous leukemia
de novo myelodysplastic syndromes
secondary myelodysplastic syndromes
previously treated myelodysplastic syndromes

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Fludarabine phosphate
Antineoplastic Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antiviral Agents
Anti-Infective Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists