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Donor Stem Cell Transplant Followed By Donor White Blood Cell Infusions in Treating Young Patients With Hematologic Cancer

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00301860
Recruitment Status : Terminated (lack of efficacy)
First Posted : March 13, 2006
Last Update Posted : July 15, 2013
National Cancer Institute (NCI)
Information provided by (Responsible Party):
University of California, San Francisco

Brief Summary:

RATIONALE: Giving chemotherapy before a donor peripheral blood stem cell transplant helps stop both the growth of cancer cells and the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving chemotherapy, such as fludarabine and melphalan, and antithymocyte globulin before transplant and cyclosporine and methotrexate after transplant may stop this from happening.

PURPOSE: This clinical trial is studying how well donor stem cell transplant, using low-dose chemotherapy and antithymocyte globulin, followed by donor white blood cell infusions work in treating young patients with hematologic cancer.

Condition or disease Intervention/treatment Phase
Leukemia Lymphoma Biological: anti-thymocyte globulin Biological: filgrastim Biological: therapeutic allogeneic lymphocytes Drug: cyclosporine Drug: fludarabine phosphate Drug: melphalan Drug: methotrexate Procedure: allogeneic hematopoietic stem cell transplantation Procedure: peripheral blood stem cell transplantation Not Applicable

Detailed Description:


  • Determine the feasibility of allogeneic hematopoietic stem cell transplantation using a reduced-intensity conditioning regimen, in terms of whole blood engraftment rate at 100 days post transplant, in pediatric patients with hematopoietic malignancies who are at high risk for complications with conventional transplantation.
  • Determine the feasibility of donor lymphocyte infusions (DLIs), in terms of number of patients who receive at least one DLI by 12 months post transplant, in patients treated with this regimen.
  • Determine the toxicities of the conditioning regimen, in terms of 100-day post transplant nonrelapse-related death rate, in these patients.
  • Determine the toxicity of DLI, in terms of acute and chronic graft-vs-host disease rate and 12-month post transplant nonrelapse-related death rate, in these patients.

OUTLINE: This is a pilot study.

  • Reduced-intensity conditioning regimen: Patients receive fludarabine IV on days -6 to -2; antithymocyte globulin IV on days -5 to -2; and melphalan IV on days -3 and -2.
  • Transplantation: Patients undergo allogeneic peripheral blood stem cell transplantation on day 0. Patients also receive filgrastim (G-CSF) IV beginning on day 5 and continuing until blood counts recover.
  • Graft-vs-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV or orally beginning on day -1 and continuing until at least day 28 and methotrexate IV on days 1, 3, and 6.
  • Donor lymphocyte infusion (DLI): Patients with mixed chimerism, no acute GVHD requiring therapy, and no relapse/progression post transplant at day 90 may receive DLI. At least 30 days after discontinuation of immunosuppression, patients may receive up to 2 DLIs at least 8-12 weeks apart in the absence of GVHD.

At the completion of study treatment, patients are followed periodically for 2 years.

PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Feasibility of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation Followed by Donor Lymphocyte Infusions for Children at High Risk for Complications With Conventional Transplantation
Study Start Date : January 2003
Actual Primary Completion Date : March 2007
Actual Study Completion Date : November 2007

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of one of the following hematopoietic malignancies:

    • Acute lymphoblastic leukemia or myeloid leukemia with < 30% blasts in the bone marrow
    • Juvenile myelomonocytic leukemia
    • Chronic myelogenous leukemia in chronic or accelerated phase
    • Relapsed non-Hodgkin's or Hodgkin's lymphoma in at least partial remission
  • Considered at high risk (> 30%) of toxic death with standard hematopoietic stem cell transplantation (HSCT), as indicated by at least one of the following:

    • Creatinine > 1.5 times normal OR creatinine clearance < 70 mL/min OR tubular damage that is not corrected by cessation of chemotherapy
    • DLCO < 60% of predicted OR history of prior intubation due to lung disease (intubation for surgery excluded)
    • Shortening fraction < 30%
    • History of disseminated fungal infection during chemotherapy OR currently receiving antifungal agents OR history of ≥ 2 septic episodes (confirmed by cultures) that required ICU support

      • Patients with improving fungal or other infections eligible

        • Improving infection is defined as confirmed negative cultures on 2 separate occasions, at least 1 week apart, and/or stable or improving imaging studies (e.g., CT scan) of the infected site
        • Two imaging studies taken at least 2 weeks apart must show stable or improved disease
    • History of stroke or abnormal MRI/MRA OR leukoencephalopathy OR seizures that are not fully controlled with anticonvulsants (> 2 episodes of seizures in the preceding year or 1 episode of status epilepticus in a patient who is receiving anticonvulsant therapy)
    • History of prior significant bleeding (e.g., pulmonary, CNS, or gastrointestinal) OR history of a clotting disorder as manifested by prior significant thromboses (e.g., superior vena cava, inferior vena cava, or femoral vein)
  • Failed conventional therapies and not eligible for myeloablative protocols

    • May have failed prior conventional HSCT
  • No active CNS leukemia
  • Unrelated or related donor available, meeting the following criteria:

    • Matched for at least 7/8 loci by high-resolution typing
    • One mismatch at A, B, or C loci allowed
    • Fully matched at DRB1 locus


  • ECOG performance status (PS) 0-2 OR Karnofsky PS 60-100%
  • No active/progressing viral, bacterial, protozoal, or fungal infection
  • Transaminases ≤ 5 times normal (except in the presence of autoimmune liver disease)
  • Shortening fraction ≥ 25%
  • DLCO ≥ 40% OR pulse oximetry ≥ 85% on room air
  • Glomerular filtration rate ≥ 40 mL/min


  • See Disease Characteristics
  • Prior prolonged intensive chemotherapy (> 3 years of therapy or ≥ 3 different chemotherapeutic protocols) allowed

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 identifier (NCT number): NCT00301860

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United States, California
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, United States, 94115
Sponsors and Collaborators
University of California, San Francisco
National Cancer Institute (NCI)
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Study Chair: Biljana Horn, MD University of California, San Francisco
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Responsible Party: University of California, San Francisco Identifier: NCT00301860    
Other Study ID Numbers: CDR0000462439
First Posted: March 13, 2006    Key Record Dates
Last Update Posted: July 15, 2013
Last Verified: July 2013
Keywords provided by University of California, San Francisco:
childhood acute lymphoblastic leukemia in remission
childhood acute myeloid leukemia in remission
recurrent childhood acute lymphoblastic leukemia
recurrent childhood acute myeloid leukemia
recurrent childhood small noncleaved cell lymphoma
juvenile myelomonocytic leukemia
chronic phase chronic myelogenous leukemia
accelerated phase chronic myelogenous leukemia
relapsing chronic myelogenous leukemia
recurrent childhood large cell lymphoma
recurrent childhood lymphoblastic lymphoma
recurrent/refractory childhood Hodgkin lymphoma
childhood chronic myelogenous leukemia
Additional relevant MeSH terms:
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Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Fludarabine phosphate
Antilymphocyte Serum
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Physiological Effects of Drugs
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists
Immunosuppressive Agents
Immunologic Factors
Antirheumatic Agents
Nucleic Acid Synthesis Inhibitors