Total-Body Irradiation With or Without Fludarabine Phosphate Followed By Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
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ClinicalTrials.gov Identifier: NCT00075478 |
Recruitment Status :
Completed
First Posted : January 12, 2004
Results First Posted : May 19, 2014
Last Update Posted : May 15, 2017
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Condition or disease | Intervention/treatment | Phase |
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Acute Lymphoblastic Leukemia in Remission Acute Myeloid Leukemia in Remission Aggressive Non-Hodgkin Lymphoma Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive Diffuse Large B-Cell Lymphoma Hematopoietic and Lymphoid Cell Neoplasm Indolent Non-Hodgkin Lymphoma Mantle Cell Lymphoma Myelodysplastic/Myeloproliferative Neoplasm Plasma Cell Myeloma Refractory Chronic Lymphocytic Leukemia Refractory Hodgkin Lymphoma Waldenstrom Macroglobulinemia | Procedure: Total-Body Irradiation Drug: Fludarabine Phosphate Drug: Mycophenolate Mofetil Drug: Cyclosporine Procedure: Peripheral Blood Stem Cell Transplantation | Phase 3 |
PRIMARY OBJECTIVES:
I. To compare overall survival at 3 years after conditioning with 200 cGy TBI alone vs. fludarabine (fludarabine phosphate)/200 cGy TBI in heavily pretreated patients with hematologic malignancies at low/moderate risk for graft rejection.
SECONDARY OBJECTIVES:
I. To compare the non-relapse mortality 1-year after conditioning in patients who received TBI alone vs. fludarabine/TBI.
II. To compare the incidences of graft rejection in patients who received TBI alone vs. fludarabine/TBI.
III. To compare the incidences of grades II-IV acute graft-versus-host disease (GVHD) and chronic extensive GVHD.
IV. To compare rates of disease progression and/or relapse-related mortality.
V. To compare the immune reconstitution and the risks of infections.
OUTLINE:
NONMYELOABLATIVE CONDITIONING REGIMEN: Patients are randomized to 1 of 2 treatment arms.
ARM I: Patients receive fludarabine phosphate intravenously (IV) on days -4 to -2. Patients then undergo low-dose TBI on day 0.
ARM II: Patients undergo low-dose TBI on day 0.
ALLOGENEIC PERIPHERAL BLOOD STEM CELL TRANSPLANTATION (PBSCT): After TBI, patients undergo PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine orally (PO) twice daily (BID) on days -3 to 56 in the absence of GVHD. Patients with no evidence of GVHD at day 56 begin a cyclosporine taper and continue the taper until day 180. Patients with evidence of disease progression and no evidence of GVHD prior to day 56 receive tapered doses of cyclosporine for 2 weeks. Patients also receive mycophenolate mofetil (MMF) PO BID on days 0-28 in the absence of GVHD. If treatment for GVHD is required before day 28, MMF is continued until a steroid taper begins.
Patients are followed up periodically for 1.5 years and then annually for 5 years post-transplantation.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 87 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Multi-center Phase III Study Comparing Nonmyeloablative Conditioning With TBI Versus Fludarabine/TBI for HLA-matched Related Hematopoietic Cell Transplantation for Treatment of Hematologic Malignancies |
Study Start Date : | October 2003 |
Actual Primary Completion Date : | February 2014 |
Actual Study Completion Date : | February 2, 2014 |

Arm | Intervention/treatment |
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Experimental: Arm I (chemotherapy, TBI, transplant, GVHD prophylaxis)
Patients receive fludarabine phosphate IV on days -4 to -2. Patients then undergo low-dose TBI on day 0. After TBI, patients undergo PBSCT on day 0. Patients receive cyclosporine PO BID on days -3 to 56 in the absence of GVHD. Patients with no evidence of GVHD at day 56 begin a cyclosporine taper and continue the taper until day 180. Patients with evidence of disease progression and no evidence of GVHD prior to day 56 receive tapered doses of cyclosporine for 2 weeks. Patients also receive MMF PO BID on days 0-28 in the absence of GVHD. If treatment for GVHD is required before day 28, MMF is continued until a steroid taper begins.
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Procedure: Total-Body Irradiation
Undergo TBI
Other Names:
Drug: Fludarabine Phosphate Given IV
Other Names:
Drug: Mycophenolate Mofetil Given PO
Other Names:
Drug: Cyclosporine Given PO
Other Names:
Procedure: Peripheral Blood Stem Cell Transplantation Undergo transplantation
Other Names:
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Active Comparator: Arm II (TBI, transplant, GVHD prophylaxis)
Patients undergo low-dose TBI on day 0. After TBI, patients undergo PBSCT on day 0. Patients receive cyclosporine PO BID on days -3 to 56 in the absence of GVHD. Patients with no evidence of GVHD at day 56 begin a cyclosporine taper and continue the taper until day 180. Patients with evidence of disease progression and no evidence of GVHD prior to day 56 receive tapered doses of cyclosporine for 2 weeks. Patients also receive MMF PO BID on days 0-28 in the absence of GVHD. If treatment for GVHD is required before day 28, MMF is continued until a steroid taper begins.
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Procedure: Total-Body Irradiation
Undergo TBI
Other Names:
Drug: Mycophenolate Mofetil Given PO
Other Names:
Drug: Cyclosporine Given PO
Other Names:
Procedure: Peripheral Blood Stem Cell Transplantation Undergo transplantation
Other Names:
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- Overall Survival [ Time Frame: 3 years after transplant ]Percentage of patients surviving as estimated by Kaplan-Meier.
- Incidence of Non-relapse Mortality [ Time Frame: 3 years after transplant ]Percentage of NRM as estimated by cumulative incidence methods with competing risks
- Incidence of Relapse/Progression [ Time Frame: 3 years after transplant ]Percentage of relapse estimated by cumulative incidence methods
- Incidence of Relapse-related Mortality [ Time Frame: 3 years after transplant ]Percentage of death following relapse/progression, estimated by cumulative incidence methods
- Incidence of Grades II-IV Acute GVHD [ Time Frame: 120 days after transplant ]Percentage patients with grades II-IV GHVD, estimated by cumulative incidence methods
- Incidence of Chronic Extensive GVHD [ Time Frame: 3 years after transplant ]Percentage patients with chronic extensive GVHD, estimated by cumulative incidence methods
- Incidence of Graft Rejection [ Time Frame: 1 year after transplant ]Donor CD3 chimerism less than 5%
- Progression-free Survival [ Time Frame: 3 years after transplant ]Percentage of patients with progression-free survival, estimated by cumulative incidence methods

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Ages Eligible for Study: | up to 75 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients must be not eligible for conventional allogeneic hematopoietic cell transplantation (HCT) and must have disease expected to be stable for at least 100 days without chemotherapy
- An autograft immediately prior (less than 6 months) to nonmyeloablative HCT (tandem approach) is not permitted
- Patients with hematologic malignancies treatable with HCT or with a B cell malignancy except those curable with autologous transplant will be included
- Aggressive non-Hodgkin lymphomas (NHLs) and other histologies such as diffuse large B cell NHL: patients are eligible IF they are not eligible for autologous hematopoietic stem cell transplantation (HSCT), not eligible for conventional myeloablative HSCT, or have failed an autologous HSCT
- Low grade NHL with < 6 month duration of complete remission (CR) between courses of conventional therapy
- Mantle cell NHL; may be treated in first CR
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Chronic lymphocytic leukemia (CLL) must have either:
- Failed to meet National Cancer Institute (NCI) Working Group criteria for complete or partial response after therapy with a regimen containing fludarabine phosphate (FLU) (or another nucleoside analog, e.g. cladribine [2-CDA], pentostatin) or experience disease relapse within 12 months after completing therapy with a regimen containing FLU (or another nucleoside analog)
- Failed FLU-cyclophosphamide [CY]-rituximab (FCR) combination chemotherapy at any time point
- Have "17p deletion" cytogenetic abnormality; patients should have received induction chemotherapy but could be transplanted in 1st CR
- Or patients with a diagnosis of CLL (or small lymphocytic lymphoma) or diagnosis of CLL that progresses to prolymphocytic leukemia (PLL), or T-cell CLL or PLL
- Hodgkin lymphoma (HL): must have received and failed frontline therapy; patients must have failed or were not eligible for autologous transplant
- Multiple myeloma (MM): must have chemosensitive disease after failed autografting (an autografting immediately prior [within 6 months] to nonmyeloablative HCT [tandem approach] is not permitted)
- Acute myeloid leukemia (AML): must have < 5% marrow blasts at the time of transplant and be beyond first CR
- Acute lymphocytic leukemia (ALL): must have < 5% marrow blasts at the time of transplant and be beyond first CR
- Chronic myelogenous leukemia (CML): patients will be accepted in chronic phase (CP) beyond CP1 if they have received previous myelosuppressive chemotherapy or HCT, < 5% marrow blasts at time of transplant
- Myelodysplastic syndromes (MDS)/myeloproliferative disorders (MPD): must have received previous myelosuppressive chemotherapy or HCT, < 5% marrow blasts at time of transplant
- Waldenstroms Macroglobulinemia: must have failed 2 courses of therapy
- Patients will not be allowed to receive myelosuppressive chemotherapy for three weeks prior to conditioning
- Patients < 12 years old must be approved by both the participating institutions' patient review committee such as the Patient Care Conference (PCC) at the Fred Hutchinson Cancer Research Center (FHCRC) and the FHCRC principal investigator
- Patients who refused to be treated on a conventional HCT protocol; for this inclusion criterion, transplants must be approved by both the participating institution's patient review committee such as the Patient Care Conference (PCC) at the FHCRC and the FHCRC principal investigator
- Patients with human leukocyte antigen (HLA)-matched related donors
- DONOR: Related donor who is HLA genotypically identical at least at one haplotype and may be phenotypically or genotypically identical at the allele level at HLA-A, -B, -C, -DRB1, and -DQB1
- DONOR: Donor must consent to filgrastim (G-CSF) administration and leukapheresis
- DONOR: Donor must have adequate veins for leukapheresis or agree to placement of central venous catheter (femoral, subclavian)
- DONOR: For females of child bearing age, serum pregnancy qualitative (PGSTAT) within 72 hours prior to initial dose of filgrastim (G-CSF); results must be available prior to filgrastim
Exclusion Criteria:
- Eligible for a high priority curative autologous transplant
- Patients with rapidly progressive, aggressive NHL unless in minimal disease state
- Patients with chronic myelomonocytic leukemia
- Presence of circulating leukemic blasts (in the peripheral blood) detected by standard pathology for patients with AML, ALL or CML
- Life expectancy severely limited by diseases other than malignancy
- Any current central nervous system (CNS) involvement with disease refractory to intrathecal chemotherapy
- Fertile men or women unwilling to use contraceptives during and for up to 12 months post treatment
- Female patients who are pregnant or breastfeeding
- Human immunodeficiency virus (HIV) positive patients
- Patients with active non-hematological malignancies (except localized non-melanoma skin malignancies)
- Patients with a history of non-hematologic malignancies (except non-melanoma skin cancers) currently in a complete remission, who are less than 5 years from the time of complete remission, and have a > 20% risk of disease recurrence
- Fungal infections with radiological progression after receipt of amphotericin formulation or mold-active azoles for greater than 1 month
- Patients with active bacterial or fungal infections unresponsive to medical therapy
- Karnofsky score < 50 for adult patients
- Lansky-Play performance score < 50 for pediatric patients
- The addition of cytotoxic agents for "cytoreduction" with the exception of tyrosine kinase inhibitors (imatinib mesylate), cytokine therapy, hydroxyurea, low dose cytarabine, chlorambucil, or rituxan will not be allowed within three weeks of the initiation of conditioning
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Patients with the following organ dysfunction:
- Symptomatic coronary artery disease or ejection fraction < 35% or other cardiac failure requiring therapy (required for patients with history of cardiac disease or anthracycline use); ejection fraction is required if age > 50 years or there is a history of anthracycline exposure or history of cardiac disease
- Poorly controlled hypertension on multiple antihypertensives
- Pulmonary: diffusion capacity of carbon monoxide (DLCO) < 30%, total lung capacity (TLC) < 30%, forced expiratory volume in one second (FEV1) < 30% and/or receiving supplementary continuous oxygen; the FHCRC study principal investigator (PI) must approve enrollment of all patients with pulmonary nodules
- Liver function abnormalities: patients with clinical or laboratory evidence of liver disease would be evaluated for the cause of liver disease, its clinical severity in terms of liver function, bridging fibrosis, and the degree of portal hypertension; patients will be excluded if they are found to have fulminant liver failure, cirrhosis of the liver with evidence of portal hypertension, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evinced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin > 3 mg/dL, and symptomatic biliary disease
- DONOR: Age less than 12 years
- DONOR: Identical twin
- DONOR: Pregnancy
- DONOR: Infection with HIV
- DONOR: Known allergy to filgrastim
- DONOR: Current serious systemic illness that would result in increased risk for filgrastim mobilization and harvest of PBSC

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): NCT00075478
United States, Oregon | |
OHSU Cancer Institute-Southern Region | |
Medford, Oregon, United States, 97504 | |
United States, Utah | |
Huntsman Cancer Institute/University of Utah | |
Salt Lake City, Utah, United States, 84112 | |
LDS Hospital | |
Salt Lake City, Utah, United States, 84143 | |
United States, Washington | |
VA Puget Sound Health Care System | |
Seattle, Washington, United States, 98101 | |
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium | |
Seattle, Washington, United States, 98109 | |
United States, Wisconsin | |
Froedtert and the Medical College of Wisconsin | |
Milwaukee, Wisconsin, United States, 53226 | |
Germany | |
Medizinische Univ Klinik Koln | |
Koln, Germany, 50924 | |
Universitaet Leipzig | |
Leipzig, Germany, D-04103 | |
University of Tuebingen-Germany | |
Tuebingen, Germany, D-72076 | |
Italy | |
University of Torino | |
Torino, Italy, 10126 |
Principal Investigator: | Brenda Sandmaier | Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium |
Responsible Party: | Brenda Sandmaier, Principal Investigator, Fred Hutchinson Cancer Research Center |
ClinicalTrials.gov Identifier: | NCT00075478 |
Other Study ID Numbers: |
1813.00 NCI-2009-01532 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ) 1813.00 ( Other Identifier: Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium ) P30CA015704 ( U.S. NIH Grant/Contract ) P01CA078902 ( U.S. NIH Grant/Contract ) |
First Posted: | January 12, 2004 Key Record Dates |
Results First Posted: | May 19, 2014 |
Last Update Posted: | May 15, 2017 |
Last Verified: | April 2017 |
Lymphoma Leukemia Neoplasms Lymphoma, Non-Hodgkin Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Lymphocytic, Chronic, B-Cell Lymphoma, Mantle-Cell Leukemia, Myelogenous, Chronic, BCR-ABL Positive Lymphoma, Large B-Cell, Diffuse Waldenstrom Macroglobulinemia Leukemia, Myeloid, Chronic-Phase Multiple Myeloma Myeloproliferative Disorders Myelodysplastic-Myeloproliferative Diseases Neoplasms by Histologic Type |
Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Leukemia, Myeloid Leukemia, Lymphoid Leukemia, B-Cell Lymphoma, B-Cell Bone Marrow Diseases Hematologic Diseases Neoplasms, Plasma Cell Hemostatic Disorders Vascular Diseases Cardiovascular Diseases Paraproteinemias |