NCT00245037

Brief Summary

RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the side effects of giving busulfan and fludarabine together with total-body irradiation and to see how well they work in treating patients who are undergoing a donor stem cell transplant for hematologic cancer.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
147

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jun 2005

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2005

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

October 25, 2005

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 27, 2005

Completed
9.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2015

Completed
1.9 years until next milestone

Results Posted

Study results publicly available

July 2, 2017

Completed
Last Updated

September 27, 2017

Status Verified

September 1, 2017

Enrollment Period

10.2 years

First QC Date

October 25, 2005

Results QC Date

April 20, 2017

Last Update Submit

September 25, 2017

Conditions

Keywords

recurrent adult acute lymphoblastic leukemiarecurrent adult acute myeloid leukemiade novo myelodysplastic syndromespreviously treated myelodysplastic syndromessecondary myelodysplastic syndromesatypical chronic myeloid leukemia, breakpoint cluster region-Abelson (BCR-ABL) negativerefractory chronic lymphocytic leukemiastage III chronic lymphocytic leukemiastage IV chronic lymphocytic leukemiaaccelerated phase chronic myelogenous leukemiablastic phase chronic myelogenous leukemiachronic phase chronic myelogenous leukemiarelapsing chronic myelogenous leukemiastage II multiple myelomastage III multiple myelomaprimary systemic amyloidosisrefractory multiple myelomamyelodysplastic/myeloproliferative neoplasm, unclassifiablechronic eosinophilic leukemiaprimary myelofibrosischronic neutrophilic leukemiaessential thrombocythemiapolycythemia verachronic myelomonocytic leukemiaacute undifferentiated leukemiaextramedullary plasmacytomaisolated plasmacytoma of bonemonoclonal gammopathy of undetermined significancestage I multiple myelomasecondary acute myeloid leukemiaadult acute myeloid leukemia with 11q23 (MLL) abnormalitiesadult acute myeloid leukemia with inv(16)(p13;q22)adult acute myeloid leukemia with t(15;17)(q22;q12)adult acute myeloid leukemia with t(16;16)(p13;q22)adult acute myeloid leukemia with t(8;21)(q22;q22)nodal marginal zone B-cell lymphomanoncontiguous stage II adult Burkitt lymphomanoncontiguous stage II adult diffuse large cell lymphomanoncontiguous stage II adult diffuse mixed cell lymphomanoncontiguous stage II adult diffuse small cleaved cell lymphomanoncontiguous stage II adult immunoblastic large cell lymphomanoncontiguous stage II adult lymphoblastic lymphomanoncontiguous stage II grade 1 follicular lymphomanoncontiguous stage II grade 2 follicular lymphomanoncontiguous stage II grade 3 follicular lymphomanoncontiguous stage II mantle cell lymphomanoncontiguous stage II marginal zone lymphomanoncontiguous stage II small lymphocytic lymphomarecurrent adult Burkitt lymphomarecurrent adult Hodgkin lymphomarecurrent adult diffuse large cell lymphomarecurrent adult diffuse mixed cell lymphomarecurrent adult diffuse small cleaved cell lymphomarecurrent adult immunoblastic large cell lymphomarecurrent adult lymphoblastic lymphomarecurrent cutaneous T-cell non-Hodgkin lymphomarecurrent grade 1 follicular lymphomarecurrent grade 2 follicular lymphomarecurrent grade 3 follicular lymphomarecurrent mantle cell lymphomarecurrent marginal zone lymphomarecurrent small lymphocytic lymphomasplenic marginal zone lymphomastage III adult Burkitt lymphomastage III adult Hodgkin lymphomastage III adult diffuse large cell lymphomastage III adult diffuse mixed cell lymphomastage III adult diffuse small cleaved cell lymphomastage III adult immunoblastic large cell lymphomastage III adult lymphoblastic lymphomastage III grade 1 follicular lymphomastage III grade 2 follicular lymphomastage III grade 3 follicular lymphomastage III mantle cell lymphomastage III marginal zone lymphomastage III small lymphocytic lymphomastage IV adult Burkitt lymphomastage IV adult Hodgkin lymphomastage IV adult diffuse large cell lymphomastage IV adult diffuse mixed cell lymphomastage IV adult diffuse small cleaved cell lymphomastage IV adult immunoblastic large cell lymphomastage IV adult lymphoblastic lymphomastage IV grade 1 follicular lymphomastage IV grade 2 follicular lymphomastage IV grade 3 follicular lymphomastage IV mantle cell lymphomastage IV marginal zone lymphomastage IV small lymphocytic lymphomaextranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue

Outcome Measures

Primary Outcomes (2)

  • Regimen-Related Toxicities

    Non-hematologic toxicities and adverse experiences ≥ Grade 3 occurrences measured up to day +100 using the NCI Common Toxicity Criteria for Adverse Events v3.0 (CTCAE). Infections and GVHD will be assessed up to 5 years post transplant. The following data represents the number of regimen-related, grade 3 and 4 toxicities that occurred in each category.

    5 years post-transplant

  • Non-relapse Mortality

    Percent of subjects with non-relapse mortality two years after conditioning with busulfan with fludarabine/200 cGy TBI in patients with hematologic malignancies at moderate to high risk for graft rejection and/or relapse of underlying disease.

    Two years post-transplant

Secondary Outcomes (5)

  • Overall Survival

    Years 1, 2, 3 and 5

  • Progression-Free Survival

    Years 1, 2, 3, and 5

  • Relapse Mortality

    Years 1 and 2

  • Acute Graft-Versus-Host Disease (aGVHD) Outcome

    Day 100, Month 6

  • Chronic Graft-Versus-Host Disease (cGVHD) Outcome

    Years 1, 2 and 3

Study Arms (1)

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

EXPERIMENTAL

Busulfan 3.2 mg/kg IV on day -5 Fludarabine 30 mg/m2/day x 3 (total dose 90 mg/m2, day -4 to day -2 TBI 200 centigray (cGy) x 1, day 0

Biological: therapeutic allogeneic lymphocytesDrug: busulfanDrug: cyclosporineDrug: fludarabine phosphateDrug: mycophenolate mofetilProcedure: peripheral blood stem cell transplantationRadiation: Total Body Irradiation (TBI)Drug: Granulocyte colony-stimulating factor (G-CSF)Drug: PhenytoinDrug: Methotrexate

Interventions

A population of lymphocytes therapeutically administered to a recipient individual who is genetically distinct from a donor of the same species.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Busulfan is an alkylating chemotherapeutic agent which has been used in many high dose and reduced intensity regimens prior to allogeneic or autologous hematopoietic stem cell transplants. It is active in a wide variety of malignancies and in high-doses it is myeloablative. IV busulfan is available and diluted and administered per package insert guidelines.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Cyclosporine is a cyclic polypeptide immunosuppressive agent. It blocks the calcium-dependent calcineurin-mediated nuclear localization of nuclear factor of activated T cells (NFAT) following T-cell activation, thereby inhibiting transactivation of key T-cell response genes including Interleukin 2 (IL-2) and Interleukin 4 (IL-4). \- Starting on day -3, Cyclosporine (CSP) is given at a dose of 4.0 mg/kg p.o. b.i.d.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Fludarabine's active metabolite 2-fluoro-ara-A is an antimetabolite that inhibits DNA primase, DNA polymerase alpha and ribonucleotide nuclease. * Dosing: Days -4, -3 and -2: Fludarabine 30 mg/m2/day IV. Total dose equals 90 mg/m2. * Monitoring: Fludarabine level is not monitored. * Dose Adjustments: There are no provisions for fludarabine dose adjustments.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Mycophenolate mofetil (MMF) is the morpholinyl ethyl ester of mycophenolic acid (MPA) and reversibly inhibits inosine monophosphate dehydrogenase, particularly the type II isoform that is more prominent in activated lymphocytes. As a result of the inhibition of de novo purine synthesis, proliferation of B- and T-lymphocytes is blocked and antibody production is inhibited. * Related Donors: MMF will be given daily at 15mg/kg q 12 hrs until day +28, then stop without tapering. Doses will be rounded to the nearest 250 mg (capsules are 250 mg). * Unrelated Donors: MMF will be given daily at 15mg/kg q 8 hrs until day +28, then given daily at 15mg/kg q 12 hours until day +56, then stop without tapering. Doses will be rounded to the nearest 250 mg (capsules are 250 mg).

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Bone marrow transplantation (BMT) and peripheral blood stem cell transplantation (PBSCT) are procedures that restore stem cells that have been destroyed by high doses of chemotherapy and/or radiation therapy. There are three types of transplants: * In autologous transplants, patients receive their own stem cells. * In syngeneic transplants, patients receive stem cells from their identical twin. * In allogeneic transplants, patients receive stem cells from their brother, sister, or parent. A person who is not related to the patient (an unrelated donor) also may be used.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

TBI is a form of radiotherapy used primarily as part of the preparative regimen for haematopoietic stem cell (or bone marrow) transplantation. As the name implies, TBI involves irradiation of the entire body, though in modern practice the lungs are often partially shielded to lower the risk of radiation-induced lung injury. * Toxicities: At the dosage used, side effects are not expected. Nevertheless, there may be fever, alopecia, parotitis, diarrhea, reversible skin pigmentation, mucositis and late effects including cataract formation, pulmonary damage, carcinogenesis, and sterilization. * Dosing: TBI will be given in one 200 cGy fraction from linear accelerator at a rate of 15-19 cGy/min.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Granulocyte colony-stimulating factor (G-CSF or GCSF) is a colony-stimulating factor hormone. G-CSF is also known as colony-stimulating factor 3 (CSF 3). It is a glycoprotein, growth factor and cytokine produced by a number of different tissues to stimulate the bone marrow to produce granulocytes and stem cells. G-CSF then stimulates the bone marrow to release them into the blood. * Toxicities: At the dosage used, the most common side effect will be medullary bone pain. * Dosing: 5 mcg/kg/day given per institutional standards (on approximately days 10-15 or not at all).

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

This drug is used to prevent seizures while on chemotherapy.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Methotrexate is used to treat severe psoriasis (a skin disease in which red, scaly patches form on some areas of the body) that cannot be controlled by other treatments.

Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
DISEASE CHARACTERISTICS: * Diagnosis of a hematologic malignancy of 1 of the following high-risk types: * Acute lymphoblastic leukemia * Acute myeloid leukemia * Chronic myelogenous leukemia * Chronic lymphocytic leukemia * Myelodysplastic syndromes * Myeloproliferative disorder * Multiple myeloma * Plasma cell dyscrasias * Non-Hodgkin lymphoma * Hodgkin disease PATIENT CHARACTERISTICS: Performance status * Karnofsky 50-100% Life expectancy * Not specified Hematopoietic * Not specified Hepatic * No liver failure * No cirrhosis with evidence of portal hypertension * No alcoholic hepatitis * No esophageal varices * No chronic hepatitis * No other liver disease Renal * Not specified Cardiovascular * Left Ventricular Ejection Fraction (LVEF) \> 35% * No symptomatic coronary artery disease or cardiac failure requiring therapy Pulmonary * Diffusing capacity of lung for carbon monoxide (DLCO) \> 30% * Total lung capacity \> 30% * Forced expiratory volume in 1 second (FEV\_1) \> 30% * No supplementary continuous oxygen Other * HIV negative * No active nonhematologic malignancy except localized skin cancer * No overt organ dysfunction PRIOR CONCURRENT THERAPY: * Not specified

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

Knight Cancer Institute at Oregon Health and Science University

Portland, Oregon, 97239-3098, United States

Location

MeSH Terms

Conditions

Myeloproliferative DisordersLeukemiaLymphomaMultiple MyelomaNeoplasms, Plasma CellMyelodysplastic SyndromesMyelodysplastic-Myeloproliferative DiseasesPrecancerous ConditionsPrecursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myeloid, AcuteLeukemia, Myeloid, Chronic, Atypical, BCR-ABL NegativeLeukemia, Lymphocytic, Chronic, B-CellLeukemia, Myeloid, Accelerated PhaseBlast CrisisLeukemia, Myeloid, Chronic-PhaseImmunoglobulin Light-chain AmyloidosisPdgfra-Associated Chronic Eosinophilic LeukemiaPrimary MyelofibrosisLeukemia, Neutrophilic, ChronicThrombocythemia, EssentialPolycythemia VeraLeukemia, Myelomonocytic, ChronicLeukemia, Biphenotypic, AcuteMonoclonal Gammopathy of Undetermined SignificanceCongenital AbnormalitiesLymphoma, B-Cell, Marginal ZoneBurkitt LymphomaHodgkin DiseaseLymphoma, Large B-Cell, DiffuseLymphoma, Non-HodgkinLymphoma, Large-Cell, ImmunoblasticLymphoma, T-Cell, CutaneousLymphoma, FollicularLymphoma, Mantle-Cell

Interventions

BusulfanCyclosporinefludarabine phosphateMycophenolic AcidPeripheral Blood Stem Cell TransplantationWhole-Body IrradiationGranulocyte Colony-Stimulating FactorPhenytoinMethotrexate

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHemorrhagic DisordersLeukemia, LymphoidLeukemia, MyeloidChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLeukemia, B-CellLeukemia, Myelogenous, Chronic, BCR-ABL PositiveCell Transformation, NeoplasticCarcinogenesisNeoplastic ProcessesAmyloidosisProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesBlood Coagulation DisordersThrombocytosisBlood Platelet DisordersBone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteHypergammaglobulinemiaCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLymphoma, B-CellEpstein-Barr Virus InfectionsHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsTumor Virus InfectionsLymphoma, T-Cell

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsCyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and ProteinsCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsHematopoietic Stem Cell TransplantationStem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, OperativeRadiotherapyInvestigative TechniquesColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsProteinsBiological FactorsHydantoinsImidazolidinesImidazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsAminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Results Point of Contact

Title
Richard T. Maziarz, MD
Organization
Oregon Health and Science University

Study Officials

  • Richard Maziarz, MD

    OHSU Knight Cancer Institute

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

October 25, 2005

First Posted

October 27, 2005

Study Start

June 1, 2005

Primary Completion

August 1, 2015

Study Completion

August 1, 2015

Last Updated

September 27, 2017

Results First Posted

July 2, 2017

Record last verified: 2017-09

Locations