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Cyclophosphamide Plus Bone Marrow Transplantation in Treating Patients With Hematologic Cancer

This study has been completed.
National Cancer Institute (NCI)
Information provided by:
Sidney Kimmel Comprehensive Cancer Center Identifier:
First received: July 5, 2000
Last updated: March 9, 2010
Last verified: March 2010

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Bone marrow transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy used to kill cancer cells.

PURPOSE: Phase I trial to study the effectiveness of cyclophosphamide plus bone marrow transplantation in treating patients who have hematologic cancer.

Condition Intervention Phase
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Biological: filgrastim
Drug: cyclophosphamide
Drug: fludarabine phosphate
Drug: mycophenolate mofetil
Drug: tacrolimus
Procedure: allogeneic bone marrow transplantation
Radiation: radiation therapy
Phase 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: Non-Myeloablative Allogeneic Bone Marrow Transplantation for Hematologic Malignancies Using Haploidentical Donors: A Phase I Trial of Pre-Transplant Cyclophosphamide

Resource links provided by NLM:

Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:

Study Start Date: December 1999
Detailed Description:


  • Determine the minimum effective dose of pretransplant cyclophosphamide to induce engraftment of haploidentical allogeneic bone marrow without the use of myeloablative conditioning in patients with hematologic malignancies.
  • Determine the incidence and severity of graft versus host disease and nonhematologic toxicities with this treatment regimen in these patients.
  • Correlate the pretreatment phenotypic and functional immunologic characteristics in these patients in relation to risk of graft rejection with this treatment regimen.

OUTLINE: This is a dose-escalation study of cyclophosphamide.

Patients receive fludarabine IV over 1 hour on days -6 to -2; cyclophosphamide IV over 1 hour on days -6, -5, and 3; total body irradiation on day -1; and allogeneic bone marrow transplantation on day 0. Patients also receive tacrolimus IV or orally twice a day on days 4-50; oral mycophenolate mofetil on days 4-35; and filgrastim (G-CSF) subcutaneously or IV starting on day 4 and continuing until blood counts recover.

Cohorts of 3-6 patients receive escalating doses of cyclophosphamide until the minimum effective dose necessary to induce chimerism without unacceptable toxicity in these patients is determined.

Patients are followed at 2 and 6 months, at one year, and then annually thereafter.

PROJECTED ACCRUAL: At least 23 patients will be accrued for this study.


Ages Eligible for Study:   up to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Patients with any of the following diagnoses:

    • Chronic myelogenous leukemia

      • Chronic phase 1

        • Failed prior interferon alfa therapy OR
        • Relapsed after prior autologous stem cell transplantation
      • Chronic phase 2
    • Acute leukemia

      • Standard risk

        • Age over 60 years
        • Complete remission 1 (CR1)
      • High risk

        • High WBC at presentation, unfavorable cytogenetics, mixed lineage, delayed response to induction chemotherapy
        • CR1
        • Complete remission 2 or higher
    • Acute lymphocytic leukemia

      • CR1 or higher
    • Myelodysplastic syndrome

      • Untreated OR
      • CR1
    • Acute myeloid leukemia in CR1
    • Chronic lymphocytic leukemia

      • Rai stage III or IV OR
      • Received prior autologous stem cell transplantation
    • Multiple myeloma

      • Stage II or III
      • Stable or progressive disease after prior chemotherapy OR
      • Received prior autologous stem cell transplantation
    • Non-Hodgkin's Lymphoma
    • Hodgkin's lymphoma
  • Ineligible for or refused autologous or standard allogeneic bone marrow transplantation
  • Ineligible for bone marrow transplantation from an HLA matched, sibling donor or from an HLA matched, unrelated donor
  • Must have an HLA mismatched, related donor (3-5 out of 6)



  • 0.5 to 70

Performance status:

  • ECOG 0-1

Life expectancy:

  • Not specified


  • Not specified


  • Bilirubin less than 3.1 mg/dL


  • Not specified


  • Left ventricular ejection fraction at least 35%


  • FEV_1 and FVC at least 40% of predicted OR
  • FEV_1 and FVC at least 60% in patients who have received prior thoracic or mantle radiotherapy


  • HIV negative
  • No other debilitating medical or psychiatric illness that would preclude study compliance
  • Not pregnant
  • Negative pregnancy test
  • Fertile patients must use effective contraception


Biologic therapy:

  • See Disease Characteristics
  • No prior transfusions from donor


  • See Disease Characteristics

Endocrine therapy:

  • Not specified


  • Not specified


  • Not specified
  Contacts and Locations
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Please refer to this study by its identifier: NCT00006042

United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231-2410
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
National Cancer Institute (NCI)
Study Chair: Ephraim J. Fuchs, MD Sidney Kimmel Comprehensive Cancer Center
  More Information

Publications: Identifier: NCT00006042     History of Changes
Other Study ID Numbers: CDR0000068057, J9966
P30CA006973 ( US NIH Grant/Contract Award Number )
Study First Received: July 5, 2000
Last Updated: March 9, 2010

Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
recurrent adult Hodgkin lymphoma
Burkitt lymphoma
refractory multiple myeloma
stage II multiple myeloma
stage III multiple myeloma
recurrent childhood lymphoblastic lymphoma
stage III chronic lymphocytic leukemia
stage IV chronic lymphocytic leukemia
relapsing chronic myelogenous leukemia
refractory chronic lymphocytic leukemia
chronic phase chronic myelogenous leukemia
adult acute myeloid leukemia in remission
adult acute lymphoblastic leukemia in remission
childhood acute myeloid leukemia in remission
childhood acute lymphoblastic leukemia in remission
recurrent/refractory childhood Hodgkin lymphoma
stage II adult lymphoblastic lymphoma
stage III grade 3 follicular lymphoma
stage III adult diffuse mixed cell lymphoma
stage III adult diffuse large cell lymphoma
stage III adult immunoblastic large cell lymphoma
stage III adult lymphoblastic lymphoma
stage III adult Burkitt lymphoma
stage IV grade 1 follicular lymphoma
stage IV grade 2 follicular lymphoma
stage IV grade 3 follicular lymphoma
stage IV adult diffuse mixed cell lymphoma
stage IV adult diffuse large cell lymphoma
stage IV adult immunoblastic large cell lymphoma
stage IV adult lymphoblastic lymphoma

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Pathologic Processes
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Bone Marrow Diseases
Precancerous Conditions
Mycophenolate mofetil processed this record on May 22, 2017