NCT00343798

Brief Summary

This phase I multicenter feasibility trial is studying the safety and potential efficacy of infusing ex vivo expanded cord blood progenitors with one unmanipulated umbilical cord blood unit for transplantation following conditioning with fludarabine, cyclophosphamide and total body irradiation (TBI), and immunosuppression with cyclosporine and mycophenolate mofetil (MMF) for patients with hematologic malignancies. Chemotherapy, such as fludarabine and cyclophosphamide, and TBI given before an umbilical cord blood transplant stops the growth of leukemia cells and works to prevent the patient's immune system from rejecting the donor's stem cells. The healthy stem cells from the donor's umbilical cord blood help the patient's bone marrow make new red blood cells, white blood cells, and platelets. It may take several weeks for these new blood cells to grow. During that period of time, patients are at increased risk for bleeding and infection. Faster recovery of white blood cells may decrease the number and severity of infections. Studies have shown that counts are more likely to recover more quickly if increased numbers of cord blood cells are given with the transplant. We have developed a way of growing or "expanding" the number of cord blood cells in the lab so that there are more cells available for transplant. We are doing this study to find out whether or not giving these expanded cells along with one unexpanded cord blood unit is safe and if use of expanded cells can decrease the time it takes for white blood cells to recover after transplant. We will study the time it takes for blood counts to recover, which of the two cord blood units makes up the patient's new blood system, and how quickly immune system cells return

Trial Health

80
On Track

Trial Health Score

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

Enrollment
23

participants targeted

Target at P25-P50 for phase_1

Geographic Reach
1 country

4 active sites

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

April 1, 2006

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

June 22, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 23, 2006

Completed
7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2013

Completed
Last Updated

February 11, 2015

Status Verified

February 1, 2015

Enrollment Period

7.3 years

First QC Date

June 22, 2006

Last Update Submit

February 10, 2015

Conditions

Accelerated Phase Chronic Myelogenous LeukemiaAcute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic SyndromeAdult Acute Lymphoblastic Leukemia in RemissionAdult Acute Myeloid Leukemia in RemissionAdult Acute Myeloid Leukemia With 11q23 (MLL) AbnormalitiesAdult Acute Myeloid Leukemia With Del(5q)Adult 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)Adult Nasal Type Extranodal NK/T-cell LymphomaAnaplastic Large Cell LymphomaChildhood Acute Lymphoblastic Leukemia in RemissionChildhood Acute Myeloid Leukemia in RemissionChildhood Burkitt LymphomaChildhood Chronic Myelogenous LeukemiaChildhood Diffuse Large Cell LymphomaChildhood Immunoblastic Large Cell LymphomaChildhood Myelodysplastic SyndromesChildhood Nasal Type Extranodal NK/T-cell LymphomaChronic Phase Chronic Myelogenous LeukemiaContiguous Stage II Adult Burkitt LymphomaContiguous Stage II Adult Diffuse Large Cell LymphomaContiguous Stage II Adult Diffuse Mixed Cell LymphomaContiguous Stage II Adult Immunoblastic Large Cell LymphomaContiguous Stage II Adult Lymphoblastic LymphomaContiguous Stage II Grade 3 Follicular LymphomaContiguous Stage II Mantle Cell Lymphomade Novo Myelodysplastic SyndromesExtranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid TissueNodal 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 Immunoblastic Large Cell LymphomaNoncontiguous Stage II Adult Lymphoblastic LymphomaNoncontiguous Stage II Grade 3 Follicular LymphomaNoncontiguous Stage II Mantle Cell LymphomaPreviously Treated Myelodysplastic SyndromesProlymphocytic LeukemiaRecurrent Adult Burkitt LymphomaRecurrent Adult Diffuse Large Cell LymphomaRecurrent Adult Diffuse Mixed Cell LymphomaRecurrent Adult Grade III Lymphomatoid GranulomatosisRecurrent Adult Immunoblastic Large Cell LymphomaRecurrent Adult Lymphoblastic LymphomaRecurrent Childhood Anaplastic Large Cell LymphomaRecurrent Childhood Grade III Lymphomatoid GranulomatosisRecurrent Childhood Large Cell LymphomaRecurrent Childhood Lymphoblastic LymphomaRecurrent Grade 1 Follicular LymphomaRecurrent Grade 2 Follicular LymphomaRecurrent Grade 3 Follicular LymphomaRecurrent Mantle Cell LymphomaRecurrent Marginal Zone LymphomaRecurrent Small Lymphocytic LymphomaRefractory AnemiaRefractory Anemia With Excess BlastsRefractory Anemia With Excess Blasts in TransformationRefractory Chronic Lymphocytic LeukemiaRefractory Multiple MyelomaSecondary Acute Myeloid LeukemiaSecondary Myelodysplastic SyndromesSplenic Marginal Zone LymphomaStage I Adult Burkitt LymphomaStage I Adult Diffuse Large Cell LymphomaStage I Adult Diffuse Mixed Cell LymphomaStage I Adult Immunoblastic Large Cell LymphomaStage I Adult Lymphoblastic LymphomaStage I Childhood Lymphoblastic LymphomaStage I Grade 3 Follicular LymphomaStage I Mantle Cell LymphomaStage II Childhood Lymphoblastic LymphomaStage III Adult Burkitt LymphomaStage III Adult Diffuse Large Cell LymphomaStage III Adult Diffuse Mixed Cell LymphomaStage III Adult Immunoblastic Large Cell LymphomaStage III Adult Lymphoblastic LymphomaStage III Childhood Lymphoblastic LymphomaStage III Grade 3 Follicular LymphomaStage III Mantle Cell LymphomaStage IV Adult Burkitt LymphomaStage IV Adult Diffuse Large Cell LymphomaStage IV Adult Diffuse Mixed Cell LymphomaStage IV Adult Immunoblastic Large Cell LymphomaStage IV Adult Lymphoblastic LymphomaStage IV Childhood Lymphoblastic LymphomaStage IV Grade 3 Follicular LymphomaStage IV Mantle Cell Lymphoma

Outcome Measures

Primary Outcomes (3)

  • Severe (grades 3 and 4) acute GVHD

    Up to day 100

  • Grade greater than or equal to 3 infusional toxicity

    Day 0

  • Graft failure as defined by failure to achieve ANC greater than or equal to 500/mm^3 of donor origin

    By day +42

Study Arms (1)

Treatment (umbilical cord blood transplant)

EXPERIMENTAL

MYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate IV over 1 hour on days -8 to -6 and cyclophosphamide IV on days -7 and -6. Patients undergo TBI BID on days -4 to -1. TRANSPLANTATION : Patients undergo double-unit umbilical cord blood transplantation comprising unmanipulated umbilical cord blood unit IV over 20-30 minutes, and 4-6 hours later patients receive ex vivo-expanded umbilical cord blood cells IV over 30 minutes on day 0. GRAFT-VERSUS-HOST-DISEASE PROPHYLAXIS: Patients receive cyclosporine IV every 8 or 12 hours on days -3 to 100, followed by a taper to at least day 180. Patients also receive MMF IV every 8 hours on days -3 to 5 and then PO, if tolerated, on days 6-30.

Drug: cyclophosphamideDrug: fludarabine phosphateDrug: cyclosporineDrug: mycophenolate mofetilOther: ex-vivo umbilical cord blood expansionProcedure: double-unit umbilical cord blood transplantationProcedure: biopsyOther: immunologic techniqueOther: diagnostic laboratory biomarker analysis

Interventions

Given IV

Also known as: CPM, CTX, Cytoxan, Endoxan, Endoxana
Treatment (umbilical cord blood transplant)

Given IV

Also known as: 2-F-ara-AMP, Beneflur, Fludara
Treatment (umbilical cord blood transplant)

Given IV

Also known as: ciclosporin, cyclosporin, cyclosporin A, CYSP, Sandimmune
Treatment (umbilical cord blood transplant)

Given IV or PO

Also known as: Cellcept, MMF
Treatment (umbilical cord blood transplant)

Undergo double-unit umbilical cord blood transplantation

Treatment (umbilical cord blood transplant)

Undergo double-unit umbilical cord blood transplantation

Treatment (umbilical cord blood transplant)
biopsyPROCEDURE

Optional correlative studies

Also known as: biopsies
Treatment (umbilical cord blood transplant)

Correlative studies

Also known as: immunological laboratory methods, laboratory methods, immunological
Treatment (umbilical cord blood transplant)

Correlative studies

Treatment (umbilical cord blood transplant)

Eligibility Criteria

Age6 Months - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Patient has no existing 0-1 HLA-A, B, C, DRB1 and DQB1 matched related donor
  • Acute Myeloid Leukemia:
  • High risk first complete remission (CR1) as evidenced by preceding myelodysplastic syndromes (MDS), high risk cytogenetics (for example, monosomy 5 or 7, or HR as defined by referring institution treatment protocol), \>= 2 cycles to obtain complete remission (CR), erythroblastic or megakaryocytic leukemia; \>= second complete remission (CR2);
  • All patients must be in CR as defined by hematologic recovery and \< 5% blasts by morphology within the bone marrow and a cellularity of \>= 15%;
  • Patients in which adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry (\< 5% blasts) and, recovery of peripheral blood counts with no circulating blasts, may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures
  • Acute lymphoblastic leukemia:
  • High risk CR1 (for example, but not limited to: t(9;22), t(1;19), t(4;11) or other mixed-lineage leukemia (MLL) rearrangements, hypodiploid);
  • \> 1 cycle to obtain CR;
  • \>= CR2;
  • All patients must be in CR as defined by hematologic recovery and \< 5% blasts by morphology within the bone marrow and a cellularity of \>= 15%;
  • Patients in which adequate marrow/biopsy specimens can not be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry (\< 5% blasts) and, recovery of peripheral blood counts with no circulating blasts, may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures
  • Chronic Myelogenous Leukemia:
  • Patients in blast crisis (BC) must receive therapy and must achieve accelerated phase (AP)/chronic phase (CP) in order to be eligible (patients who remain in BC are not eligible);
  • If in first chronic phase, patient must have failed or be intolerant to imatinib mesylate
  • Myelodysplasia (MDS):
  • +14 more criteria

You may not qualify if:

  • Acute leukemia in relapse (\>= 5% marrow blasts by morphology)
  • Active central nervous system (CNS) leukemia involvement at the time of study enrollment (cerebrospinal fluid with \> 5 white blood cells (WBC)/mm\^3 AND malignant cells on cytospin)
  • Chemotherapy refractory large cell lymphoma and high grade NHL (progressive disease after \> 2 salvage regimens)
  • Female patients who are pregnant or breastfeeding
  • Karnofsky performance status \< 70% (adults) or Lanksy score \< 50% (pediatrics)
  • Prior autologous or allogeneic stem cell transplant with myeloablative preparative regimen (If =\< 18 years old, prior myeloablative transplant within the last 6 months)
  • Uncontrolled viral, or bacterial infection at the time of study enrollment
  • Active or recent (prior 6 months) invasive fungal infection without ID consult and approval
  • Seropositive for human immunodeficiency virus (HIV)
  • Consenting 5 of 6 or 6 of 6 HLA-matched related donor available
  • Unable to provide informed consent
  • Use of any other experimental drug within 28 days of baseline

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

City of Hope Medical Center

Duarte, California, 91010, United States

Location

University of Colorado Cancer Center - Anschutz Cancer Pavilion

Aurora, Colorado, 80045, United States

Location

University of Colorado

Denver, Colorado, 80217-3364, United States

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Accelerated PhaseCongenital AbnormalitiesLymphoma, Extranodal NK-T-CellLymphoma, Large-Cell, AnaplasticBurkitt LymphomaLymphoma, Large B-Cell, DiffuseLeukemia, Myeloid, Chronic-PhaseLymphoma, B-Cell, Marginal ZoneLeukemia, ProlymphocyticLymphoma, Non-HodgkinLymphoma, Large-Cell, ImmunoblasticPrecursor Cell Lymphoblastic Leukemia-LymphomaDendritic Cell Sarcoma, InterdigitatingLymphoma, FollicularLymphoma, Mantle-CellLeukemia, Lymphocytic, Chronic, B-CellAnemia, RefractoryAnemia, Refractory, with Excess of BlastsMultiple Myeloma

Interventions

Cyclophosphamidefludarabine phosphateCyclosporineMycophenolic AcidBiopsyImmunologic Techniques

Condition Hierarchy (Ancestors)

Leukemia, Myelogenous, Chronic, BCR-ABL PositiveLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLymphoma, T-CellLymphomaLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesEpstein-Barr Virus InfectionsHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsTumor Virus InfectionsLymphoma, B-CellLeukemia, LymphoidHistiocytic Disorders, MalignantHistiocytosisLeukemia, B-CellAnemiaMyelodysplastic SyndromesNeoplasms, Plasma CellHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHemorrhagic Disorders

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsCyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and ProteinsCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsCytodiagnosisCytological TechniquesClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisSpecimen HandlingDiagnostic Techniques, SurgicalSurgical Procedures, OperativeInvestigative Techniques

Study Officials

  • Colleen Delaney

    Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 22, 2006

First Posted

June 23, 2006

Study Start

April 1, 2006

Primary Completion

July 1, 2013

Last Updated

February 11, 2015

Record last verified: 2015-02

Locations