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Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

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ClinicalTrials.gov Identifier: NCT00723099
Recruitment Status : Recruiting
First Posted : July 28, 2008
Last Update Posted : January 19, 2018
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Fred Hutchinson Cancer Research Center

Brief Summary:
This phase II trial is studying how well umbilical cord blood transplant from a donor works in treating patients with hematological cancer. Giving chemotherapy and total-body irradiation (TBI) before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from an unrelated donor, that do not exactly match the patient's blood, 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 (called graft-versus-host disease). Giving cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening.

Condition or disease Intervention/treatment Phase
Acute Lymphoblastic Leukemia Acute Myeloid Leukemia Aggressive Non-Hodgkin Lymphoma Chronic Myelogenous Leukemia, BCR-ABL1 Positive Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive Indolent Non-Hodgkin Lymphoma Lymphoma Mixed Phenotype Acute Leukemia Myelodysplastic Syndrome Myeloproliferative Neoplasm Recurrent Chronic Lymphocytic Leukemia Recurrent Follicular Lymphoma Recurrent Lymphoplasmacytic Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Marginal Zone Lymphoma Recurrent Plasma Cell Myeloma Recurrent Small Lymphocytic Lymphoma Recurrent T-Cell Non-Hodgkin Lymphoma Refractory Chronic Lymphocytic Leukemia Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive Refractory Follicular Lymphoma Refractory Hodgkin Lymphoma Refractory Lymphoplasmacytic Lymphoma Refractory Mantle Cell Lymphoma Refractory Small Lymphocytic Lymphoma T-Cell Non-Hodgkin Lymphoma Procedure: Allogeneic Hematopoietic Stem Cell Transplantation Drug: Cyclophosphamide Drug: Cyclosporine Drug: Fludarabine Phosphate Other: Laboratory Biomarker Analysis Drug: Mycophenolate Mofetil Radiation: Total-Body Irradiation Procedure: Umbilical Cord Blood Transplantation Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 76 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Reduced-Intensity Preparative Regimen (A Multi-Center Trial Coordinated by the FHCRC)
Actual Study Start Date : June 25, 2008
Estimated Primary Completion Date : November 13, 2018
Estimated Study Completion Date : November 13, 2019

Arm Intervention/treatment
Experimental: Treatment (chemotherapy, transplant)

CONDITIONING REGIMEN: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1-2 hours on day -6. Patients undergo a lower dose of TBI on day -1.

UMBILICAL CORD BLOOD TRANSPLANT: Patients undergo donor umbilical cord blood infusion on day 0.

IMMUNOSUPRESSIVE THERAPIES: Patients receive cyclosporine IV over 1 hour every 8-12 hours on days 0 to +180 and mycophenolate mofetil IV or PO every 8 hours on days -3 to +96.

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo umbilical cord blood transplant
Other Names:
  • Allogeneic Hematopoietic Cell Transplantation
  • Allogeneic Stem Cell Transplantation
  • HSC
  • HSCT
Drug: Cyclophosphamide
Given IV
Other Names:
  • (-)-Cyclophosphamide
  • 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate
  • Carloxan
  • Ciclofosfamida
  • Ciclofosfamide
  • Cicloxal
  • Clafen
  • Claphene
  • CP monohydrate
  • CTX
  • CYCLO-cell
  • Cycloblastin
  • Cycloblastine
  • Cyclophospham
  • Cyclophosphamid monohydrate
  • Cyclophosphamidum
  • Cyclophosphan
  • Cyclophosphane
  • Cyclophosphanum
  • Cyclostin
  • Cyclostine
  • Cytophosphan
  • Cytophosphane
  • Cytoxan
  • Fosfaseron
  • Genoxal
  • Genuxal
  • Ledoxina
  • Mitoxan
  • Neosar
  • Revimmune
  • Syklofosfamid
  • WR- 138719
Drug: Cyclosporine
Given IV
Other Names:
  • 27-400
  • Ciclosporin
  • CsA
  • Cyclosporin
  • Cyclosporin A
  • Gengraf
  • Neoral
  • OL 27-400
  • Sandimmun
  • Sandimmune
  • SangCya
Drug: Fludarabine Phosphate
Given IV
Other Names:
  • 2-F-ara-AMP
  • 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-
  • Beneflur
  • Fludara
  • SH T 586
Other: Laboratory Biomarker Analysis
Correlative studies
Drug: Mycophenolate Mofetil
Given IV or PO
Other Names:
  • Cellcept
  • MMF
Radiation: Total-Body Irradiation
Undergo TBI
Other Names:
  • Total Body Irradiation
  • Whole-Body Irradiation
Procedure: Umbilical Cord Blood Transplantation
Undergo umbilical cord blood transplant
Other Names:
  • Cord Blood Transplantation
  • UCB transplantation

Primary Outcome Measures :
  1. Overall survival [ Time Frame: At 1 year ]
    Kaplan-Meier and cumulative incidence estimates will be used.

Secondary Outcome Measures :
  1. Incidence of chronic GVHD for each arm [ Time Frame: At 1 year ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  2. Incidence of clinically significant infections [ Time Frame: Up to 2 years ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  3. Incidence of grade II-IV and III-IV acute graft versus host disease (GVHD) for each arm [ Time Frame: By day 100 ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  4. Incidence of graft failure/rejection [ Time Frame: By day 55 ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  5. Incidence of neutrophil engraftment [ Time Frame: By day 42 ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  6. Incidence of non-relapse mortality [ Time Frame: 6 months ]
    Kaplan-Meier and cumulative incidence estimates will be used

  7. Incidence of non-relapse mortality [ Time Frame: 1 year ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  8. Incidence of non-relapse mortality [ Time Frame: Up to 2 years ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  9. Incidence of platelet engraftment [ Time Frame: By 6 months ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  10. Incidence of relapse or disease progression [ Time Frame: Up to 2 years ]
    Kaplan-Meier and cumulative incidence estimates will be used.

  11. Overall survival [ Time Frame: 2 years ]
    Kaplan-Meier and cumulative incidence estimates will be used

  12. Progression-free survival [ Time Frame: Up to 2 years ]
    Kaplan-Meier and cumulative incidence estimates will be used

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 69 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients > 70 may be considered if performance status > 80% or Eastern Cooperative Oncology Group (ECOG) =< 1 and comorbidity score < 3; these patients must be discussed with the principal investigator (PI), Rachel Salit prior to enrollment
  • Adequate cardiac function defined as absence of decompensated congestive heart failure, or uncontrolled arrhythmia and:

    • Left ventricular ejection fraction >= 35% or
    • Fractional shortening > 22%
  • Adequate pulmonary function defined as diffusion capacity of carbon monoxide (DLCO) > 30% predicted, and absence of oxygen (O2) requirements
  • Adequate hepatic function; patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, histology, and the degree of portal hypertension; patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, or correctable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin > 3 mg/dL, and symptomatic biliary disease will be excluded
  • Adequate renal function defined as creatinine =< 2.0 mg/dl (adults) or creatinine clearance > 40 ml/min (pediatrics)
  • All adults with a creatinine > 1.2 or a history of renal dysfunction must have estimated creatinine clearance > 40 ml/min
  • Performance status score: Karnofsky (for adults) >= 60 or ECOG 0-2; Lansky (for children) score >= 50
  • If recent mold infection, e.g., Aspergillus, must be cleared by infectious disease
  • Second hematopoietic cell transplant: Must be >= 3 months after prior myeloablative transplant
  • Patients who have received < 2 cycles of multiagent chemotherapy and patients who have received no multiagent chemotherapy within the 3 months previous to umbilical cord blood transplant (UCBT) as well as patients experiencing graft failure following previous allogeneic transplant
  • Acute myeloid leukemia/acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia: Must have < 5% morphologic marrow blasts in an evaluable marrow (> 25% of normal cellularity for age) collected less than one month prior to start of conditioning; patients persistently aplastic for greater than one month since completing last chemotherapy are also eligible with the approval of the PI or designee
  • Chronic myelogenous leukemia: All types, except refractory blast crisis; chronic phase patients must have failed or been intolerant to Gleevec or other tyrosine kinase inhibitors; at time of transplant, patients must have < 5% blasts in an evaluable marrow (> 25% of normal cellularity for age) by morphology within the bone marrow
  • Myelodysplastic syndrome (MDS): Any subtype; morphologic blasts must be less than 5% in an evaluable marrow (> 25% of normal cellularity for age); if blasts are 5% or more, patient requires induction chemotherapy pre-transplant to reduce blast count to less than 5%; patients who have a hypocellular marrow in the absence of excess blasts that is related to the underlying disease or as a result of treatment for MDS may also be eligible with the approval of the PI or designee
  • Large-cell lymphoma and aggressive T-cell lymphoma: With chemotherapy sensitive disease that has failed autologous transplant or patients who are ineligible for an autologous transplant; chemotherapy sensitive disease is defined as >= 50% reduction in the size of the tumor with the chemotherapy regimen immediately preceding transplant
  • Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL): Must be refractory to fludarabine (fludarabine phosphate) or fail to have a complete or partial response after therapy with a regimen containing fludarabine (or another nucleoside analog, e.g. cladribine [2-CDA], pentostatin) or experience disease relapse within 12 months after completing therapy with a regimen containing fludarabine (or another nucleoside analog)
  • Hodgkin disease: Must have received and failed frontline therapy
  • Follicular lymphoma, marginal zone B-cell lymphoma, lymphoplasmacytic lymphoma, mantle-cell lymphoma, and indolent T-cell lymphomas: Must have progressed with the most recent remission duration being < 6 months; patients with bulky disease should be considered for debulking chemotherapy before transplant; patients with refractory disease are eligible, unless they have bulky disease and an estimated tumor doubling time of less than one month
  • Multiple myeloma: Must have received prior chemotherapy; consolidation of chemotherapy by autografting prior to nonmyeloablative hematopoietic cell transplant (HCT) is permitted
  • Myeloproliferative syndromes
  • DONOR: Cord blood (CB) donor selection will be based on institutional guidelines and in general should be selected to optimize both human leukocyte antigen (HLA) match and cell dose; additionally, CB grafts shall consist of one or two CB donors based on, but not exclusively determined by, cell dose (total nucleated cell [TNC]/kg and CD34/kg), HLA matching and disease status and indication for transplant; attending preference will be allowed for single versus double unit as well as the degree of mismatching based on patient specific factors, as long as the following minimum criteria are met:

    • HLA matching

      • Minimum requirement: The CB graft(s) must be matched at a minimum at 4/6 HLA-A, B, DRB1 loci with the recipient. Therefore 0-2 mismatches at the A or B or DRB1 loci based on intermediate resolution A, B antigen and DRB1 allele typing for determination of HLA-match is allowed
      • HLA-matching determined by high-resolution typing is allowed per institutional guidelines as long as the minimum criteria are met
    • Selection of two CB units is mandatory when a single cord blood unit does not meet the following criteria:

      • Match grade 6/6; TNC Dose >= 2.5 x 10^7/kg
      • Match grade 5/6 or 4/6; TNC dose >= 4.0 (+/- 0.5) x 10^7/kg
    • If two CB units are used, the total cell dose of the combined units must be at least 3.0 x 10^7 TNC per kilogram recipient weight based on pre-cryopreservation numbers, with each CB unit containing a MINIMUM of 1.5 x 10^7 TNC/kg
    • The minimum recommended CD34/kg cell dose should be 2 x 10^5 CD34/kg, total dose from a single or combined double
    • The unmanipulated CB unit(s) will be Food and Drug Administration (FDA) licensed or will be obtained under a separate investigational new drug (IND), such as the National Marrow Donor Program (NMDP) Protocol 10-CBA conducted under BB IND-7555 or another IND sponsored by (1) a participating institution or (2) an investigator at FHCRC or one of the participating institutions
    • FHCRC only: Up to 5% of cord blood product, when ready for infusion, may be withheld for research purposes as long as thresholds for infused TNC dose are met; threshold for double unit transplantation is >= 3.0 x 10^7/kg; these products will be used to conduct studies involving the immunobiology of double cord transplantation and kinetics of engraftment

Exclusion Criteria:

  • Patients with an available 5-6/6 HLA-A, B, DRB1 matched sibling donor
  • Pregnancy or breastfeeding
  • Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology
  • Uncontrolled viral or bacterial infection at the time of study enrollment
  • Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
  • Active central nervous system malignancy
  • Patients who have received >= 2 cycles of multiagent chemotherapy within the 3 months previous to UCBT; patients who have had previous autologous transplant within 12 months of UCBT are excluded regardless of history of recent treatment
  • DONOR: Any cord blood units with < 1.5 x 10^7 total nucleated cells per kilogram recipient weight
  • DONOR: Any cord blood units without the full maternal testing and negative results for hepatitis A, B, C, HIV, and human T-lymphotropic virus (HTLV-1) viruses; any additional available virology results on the unit itself will be reviewed but are not mandated, complete or always available; cord blood units are presumed to be cytomegalovirus (CMV) negative regardless of serologic testing due to passive transmission of maternal CMV antibodies

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 ClinicalTrials.gov identifier (NCT number): NCT00723099

United States, Colorado
University of Colorado Hospital Completed
Aurora, Colorado, United States, 80045
Colorado Blood Cancer Institute Completed
Denver, Colorado, United States, 80218
United States, Utah
LDS Hospital Terminated
Salt Lake City, Utah, United States, 84143
United States, Washington
VA Puget Sound Health Care System Suspended
Seattle, Washington, United States, 98101
Fred Hutch/University of Washington Cancer Consortium Recruiting
Seattle, Washington, United States, 98109
Contact: Rachel B. Salit    206-667-1317    rsalit@fredhutch.org   
Principal Investigator: Rachel B. Salit         
United States, Wisconsin
Froedtert and the Medical College of Wisconsin Terminated
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
National Cancer Institute (NCI)
Principal Investigator: Rachel Salit Fred Hutch/University of Washington Cancer Consortium

Responsible Party: Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00723099     History of Changes
Other Study ID Numbers: 2239.00
NCI-2009-01551 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2239.00 ( Other Identifier: Fred Hutch/University of Washington Cancer Consortium )
P30CA015704 ( U.S. NIH Grant/Contract )
First Posted: July 28, 2008    Key Record Dates
Last Update Posted: January 19, 2018
Last Verified: January 2018

Additional relevant MeSH terms:
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid, Acute
Lymphoma, Follicular
Myelodysplastic Syndromes
Lymphoma, Non-Hodgkin
Leukemia, Lymphoid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myeloid
Hodgkin Disease
Lymphoma, Mantle-Cell
Lymphoma, B-Cell, Marginal Zone
Lymphoma, T-Cell
Myeloproliferative Disorders
Leukemia, Myeloid, Chronic-Phase
Multiple Myeloma
Neoplasms, Plasma Cell
Waldenstrom Macroglobulinemia
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions