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NCT00719888 · REGISTRY CONDITION SEARCH

Umbilical Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With Hematologic Disease

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE2
Status at capture
COMPLETED
Registry last update
2025-03-07

This phase II trial studies how well giving an umbilical cord blood transplant together with cyclophosphamide, fludarabine, and total-body irradiation (TBI) works in treating patients with hematologic disease. Giving chemotherapy, such as cyclophosphamide and fludarabine, and 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 a donor 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. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Overall Survival

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outcome · POSTED

Change in Level of Chimerism at Multiple Time Points

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outcome · POSTED

Incidence of Transplant-related Mortality (TRM)

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outcome · POSTED

Neutrophil Engraftment

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outcome · POSTED

Platelet Engraftment

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outcome · POSTED

Event of Grade II-IV and III-IV Acute Graft-versus-host Disease (aGVHD)

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outcome · POSTED

Event of Chronic Graft-verses-host-disease (cGVHD)

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outcome · POSTED

Event of Clinically Significant Infections

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outcome · POSTED

Incidence of Relapse

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outcome · POSTED

Progression-free Survival (PFS)

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * GRAFT CRITERIA: * UCB units will be selected according to current umbilical cord blood graft selection algorithm; one or 2 UCB units may be used to achieve the required cell dose * The UCB graft is matched at 4-6 human leukocyte antigen (HLA)-A, B, DRB1 antigens with the recipient; this may include 0-2 antigen mismatches at the A or B or DRB1 loci; unit selection based on cryopreserved nucleated cell dose and HLA-A,B, DRB1 using intermediate resolution A, B antigen and DRB1 allele typing * If 2 UCB units are required to reach the target cell dose, each unit must be a 4-6 antigen match to the recipient * Age and Disease Criteria: * High-dose TBI regimen: 6 months to =\< 45 years * Middle-intensity TBI regimen: 6 months to =\< 65 years * Conditioning regimen selection should be based on the underlying disease, presence of minimum residual disease (MRD), age, co-morbidities, and attending physician * Acute myeloid leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia: * All patients must be in complete remission (CR) as defined by hematologic recovery and \< 5% blasts by morphology/flow cytometry in a representative bone marrow sample with cellularity \>= 15% for age; patients who do not have high-risk features (for example preceding myelodysplastic syndrome \[MDS\], high-risk cytogenetics, \>= 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia or \>= CR2) must be discussed with the principal investigator (PI) prior to enrollment and at the Patient Care Conference or equivalent group such as the pediatric leukemia board as an alternative * Patients in whom adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the PI prior to enrollment; patients persistently aplastic for greater than one month since completing last chemotherapy are also eligible with PI approval * Very high risk pediatric/young adult patients with acute myeloid leukemia (AML): Patients =\< 25 years, however, are eligible with (M2 marrow) with =\< 25% blasts in marrow after having failed one or more cycles of chemotherapy; this group of patients will be analyzed separately * Acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia: * All patients must be in CR as defined by \< 5% blasts by morphology; flow cytometry in a representative bone marrow sample with cellularity \>= 15% for age; patients who do not have high-risk disease (high risk CR1, greater than one cycle to obtain CR or \>= CR2) must be discussed with the PI prior to enrollment and at the Patient Care Conference or equivalent group such as the pediatric leukemia board as an alternative * Patients in whom adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the principal investigator Ann Dahlberg prior to enrollment; patients persistently aplastic for greater than one month since completing last chemotherapy are also eligible with PI approval * Chronic myelogenous leukemia excluding refractory blast crisis; to be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate * Advanced myelofibrosis * Myelodysplasia (MDS) International Prognostic Scoring System (IPSS) intermediate (Int)-2 or high risk (i.e., refractory anemia with excess blasts \[RAEB\], RAEB in transformation \[RAEBt\]) or refractory anemia with severe pancytopenia or high risk cytogenetics; blasts must be \< 10% by a representative bone marrow aspirate morphology * Lymphoblastic lymphoma, Burkitt's lymphoma, and other high-grade non-Hodgkin lymphoma (NHL) after initial therapy if stage III/IV in first partial response (PR1) or after progression if stage I/II \< 1 year; stage III/IV patients are eligible after progression in CR/PR * Chronic lymphocytic leukemia /small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, lymphoplasmacytic lymphoma or follicular lymphoma that have progressed after at least two different prior therapies; patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant; these patients must be presented at primary care center (PCC) prior to enrollment, given potential competing eligibility on autotransplant protocols * Mantle-cell lymphoma, prolymphocytic leukemia: Eligible after initial therapy in \>= CR1 or \>= PR1 * Large cell NHL \> CR2/\> second partial response (PR2): * Patients in CR2/PR2 with initial short remission (\< 6 months) are eligible * These patients must be presented at PCC prior to enrollment, given potential competing eligibility on autotransplant protocols * Multiple myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or beta-2 microglobulin \> 3 mg/L, may be considered for this protocol after initial therapy * Performance status score: Karnofsky (for adults) \>= 70% or Eastern Cooperative Oncology Group (ECOG) 0-1 or Lansky (for children) \>= 50% * Creatinine \< 2.0 mg/dL (for adults) or creatinine clearance \> 60 ml/min (for children) * 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 * Diffusion capacity for carbon monoxide corrected (DLCOcorr) \> 50% normal or a pediatric patient who is unable to perform pulmonary function tests (PFTs) but has adequate pulmonary function * Left ventricular ejection fraction \> 45% or shortening fraction \> 26% Exclusion Criteria: * Uncontrolled viral or bacterial infection at the time of study enrollment * Active or recent (prior 6 month) invasive fungal infection without interdisciplinary (ID) consult and approval * History of human immunodeficiency virus (HIV) infection * Pregnant or breastfeeding * Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after \> 2 salvage regimens) * Patients with history of prior myeloablative transplant containing full dose TBI (greater than 8 gray \[Gy\]) will not be eligible for Regimen A; however, they may still enroll on Regimen B if they otherwise meet inclusion and exclusion criteria * Any prior myeloablative transplant within the last 6 months * Patients \>= 45 years: comorbidity score of 5 or higher * Patients who have received Y-90 ibritumomab (Zevalin) or I-131 tostumomab (Bexxar), as part of their salvage therapy are not eligible for Regimen A
healthy Volunteers
false
maximum Age
65 Years
minimum Age
6 Months
sex
ALL
std Ages
  1. CHILD
  2. ADULT
  3. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Patients receive myeloablative conditioning comprising fludarabine IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 and -6, and undergo high-dose TBI BID on days -4 to -1. Patients then undergo single- or double-unit UCBT on day 0. Patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour every 8 or 12 hours, then cyclosporine PO (if tolerated), on days -3 to 100 with taper on day 101. Patients also receive mycophenolate mofetil IV every 8 hours on days 0 to 7 and then PO (if tolerated) TID on days 8-30. Mycophenolate mofetil is tapered to BID on day 30 or 7 days after engraftment if there is no acute GVHD, and then tapered over 2-3 weeks beginning on day 45 (or 15 days after engraftment if engraftment occurred \> day 30) after engraftment if there continues to be no evidence of acute GVHD.
    intervention Names
    1. Drug: Cyclophosphamide
    2. Drug: Cyclosporine
    3. Procedure: Double-Unit Umbilical Cord Blood Transplantation
    4. Drug: Fludarabine
    5. Other: Laboratory Biomarker Analysis
    6. Drug: Mycophenolate Mofetil
    7. Radiation: Total-Body Irradiation
    8. Procedure: Umbilical Cord Blood Transplantation
    label
    Treatment (myeloablative UCBT)
    type
    EXPERIMENTAL
  2. description
    Patients receive myeloablative conditioning comprising fludarabine IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 2-4 hours on days -5 and -4, and middle-intensity TBI QD on days -2 and -1. Patients then undergo single- or double-unit UCBT on day 0. Patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour every 8 or 12 hours, then cyclosporine PO (if tolerated), on days -3 to 100 with taper on day 101. Patients also receive mycophenolate mofetil IV every 8 hours on days 0 to 7 and then PO (if tolerated) TID on days 8-30. Mycophenolate mofetil is tapered to BID on day 30 or 7 days after engraftment if there is no acute GVHD, and then tapered over 2-3 weeks beginning on day 45 (or 15 days after engraftment if engraftment occurred \> day 30) after engraftment if there continues to be no evidence of acute GVHD.
    intervention Names
    1. Drug: Cyclophosphamide
    2. Drug: Cyclosporine
    3. Procedure: Double-Unit Umbilical Cord Blood Transplantation
    4. Drug: Fludarabine
    5. Other: Laboratory Biomarker Analysis
    6. Drug: Mycophenolate Mofetil
    7. Radiation: Total-Body Irradiation
    8. Procedure: Umbilical Cord Blood Transplantation
    9. Drug: Thiotepa
    label
    Arm II (myeloablative UCBT)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Given IV
    name
    Cyclophosphamide
    other Names
    1. (-)-Cyclophosphamide
    2. 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate
    3. Carloxan
    4. Ciclofosfamida
    5. Ciclofosfamide
    6. Cicloxal
    7. Clafen
    8. Claphene
    9. CP monohydrate
    10. CTX
    11. CYCLO-cell
    12. Cycloblastin
    type
    DRUG
  2. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Given IV and PO
    name
    Cyclosporine
    other Names
    1. 27-400
    2. Ciclosporin
    3. CsA
    4. Cyclosporin
    5. Cyclosporin A
    6. Gengraf
    7. Neoral
    8. OL 27-400
    9. Sandimmun
    10. Sandimmune
    11. SangCya
    type
    DRUG
  3. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Undergo double-unit UCBT
    name
    Double-Unit Umbilical Cord Blood Transplantation
    type
    PROCEDURE
  4. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Given IV
    name
    Fludarabine
    other Names
    1. 118218
    2. Fluorovidarabine
    type
    DRUG
  5. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Correlative studies
    name
    Laboratory Biomarker Analysis
    type
    OTHER
  6. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Given IV and PO
    name
    Mycophenolate Mofetil
    other Names
    1. Cellcept
    2. MMF
    type
    DRUG
  7. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Undergo high-dose or moderate-intensity TBI
    name
    Total-Body Irradiation
    other Names
    1. Total Body Irradiation
    2. Whole-Body Irradiation
    3. SCT_TBI
    4. TBI
    type
    RADIATION
  8. arm Group Labels
    1. Arm II (myeloablative UCBT)
    2. Treatment (myeloablative UCBT)
    description
    Undergo UCBT
    name
    Umbilical Cord Blood Transplantation
    other Names
    1. Cord Blood Transplantation
    2. UCB transplantation
    type
    PROCEDURE
  9. arm Group Labels
    1. Arm II (myeloablative UCBT)
    description
    Given IV
    name
    Thiotepa
    other Names
    1. 1,1',1"-phosphinothioylidynetrisaziridine
    2. 1,1',1''-Phosphinothioyldynetrisaziridine
    3. 52-24-4
    4. 6396
    5. Girostan
    6. Triethylenethiophosphoramide
    7. Triethylene Thiophosphoramide
    type
    DRUG
Study design
allocation
NON_RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
135
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Overall survival defined as patient being alive at 1 year post-transplant. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Overall Survival
    time Frame
    1 year post-transplant
secondary Outcomes
  1. description
    Peripheral blood chimerism studies (sorted for CD3, CD14, CD33, CD56 cells) were collected for all UCBT recipients for days 28, 56, 80, 365, and 730 post-transplant. The combined median of the percent donor, along with full range, is captured to show change in level of chimerism at multiple time points up to 2 years post-transplant. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Change in Level of Chimerism at Multiple Time Points
    time Frame
    Baseline up to 2 years post-transplant
  2. description
    Defined as death due to complication (other than relapse) within 6 months following UCBT. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Incidence of Transplant-related Mortality (TRM)
    time Frame
    At 6 months post-transplant
  3. description
    Neutrophil engraftment after UCBT is defined as the first day of absolute neutrophil count (ANC) ≥ 5 x 10⁸/L for 3 consecutive measures. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Neutrophil Engraftment
    time Frame
    Up to day 42 post-transplant
  4. description
    Platelet (PLT) engraftment after UCBT is defined as the first day of platelet count \> 20,000/μl without subsequent transfusions for 7 days. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Platelet Engraftment
    time Frame
    Up to 6 months post-transplant
  5. description
    Each event of aGVHD will be assigned an overall aGVHD score based on extent of skin rash, volume of diarrhea, and maximum bilirubin level, per protocol Appendix I, "GVHD Staging and Grading." Scores reported range from most mild at 2, to worse at 4. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Event of Grade II-IV and III-IV Acute Graft-versus-host Disease (aGVHD)
    time Frame
    Up to day 100 post-transplant
  6. description
    Each event of cGVHD level will be assessed overall as either 'Limited' (mild and with single organ involvement) or 'Extensive' (involvement of two or more organs with symptoms) based on extent of skin rash, volume of diarrhea, and maximum bilirubin level, per protocol Appendix I, "GVHD Staging and Grading." Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Event of Chronic Graft-verses-host-disease (cGVHD)
    time Frame
    Up to 2 years post-transplant
  7. description
    Each event of infection will be assessed to determine whether or not it is clinically significant. An infection is defined as clinically significant when it is scored as ≥ grade 3 (where worse outcomes are associated with higher grading) in accordance with the CTCAE version 3.0/protocol Appendix VI, and constitutes a Serious Adverse Event (SAE). Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Event of Clinically Significant Infections
    time Frame
    Up to 2 years post-transplant
  8. description
    Relapse is defined as post-transplant disease recurrence in participants who had initially recovered or improved, with incidence measured from Day 0 to 1 year post-transplant, and from 1 year post-transplant to 2 years post-transplant. Monitoring will take place separately for the single and double UCBT cohorts.
    measure
    Incidence of Relapse
    time Frame
    At 1 and 2 years post-transplant
  9. description
    PFS is defined as the time (in days) from UCBT until the disease progresses or the patient dies from any cause, with incidence measured up to 2 years post-transplant. Monitoring will take place separately for the single and double UCBT cohorts. Measure will be reported as the median amount of days from full minimum and maximum range.
    measure
    Progression-free Survival (PFS)
    time Frame
    Up to 2 years post-transplant
Full study description
brief Summary
This phase II trial studies how well giving an umbilical cord blood transplant together with cyclophosphamide, fludarabine, and total-body irradiation (TBI) works in treating patients with hematologic disease. Giving chemotherapy, such as cyclophosphamide and fludarabine, and 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 a donor 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. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.
detailed Description
OUTLINE: Patients are assigned to 1 of 2 arms. ARM I: Patients receive myeloablative conditioning comprising fludarabine intravenously (IV) over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 and -6, and undergo high-dose TBI twice daily (BID) on days -4 to -1. Patients then undergo single- or double-unit UCBT on day 0. ARM II: Patients receive myeloablative conditioning comprising fludarabine IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 2-4 hours on days -5 and -4, and middle-intensity TBI once daily (QD) on days -2 and -1. Patients then undergo single- or double-unit UCBT on day 0. Patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour every 8 or 12 hours, then cyclosporine orally (PO) (if tolerated), on days -3 to 100 with taper on day 101. Patients also receive mycophenolate mofetil IV every 8 hours on days 0 to 7 and then PO (if tolerated) three times daily (TID) on days 8-30. Mycophenolate mofetil is tapered to BID on day 30 or 7 days after engraftment if there is no acute GVHD, and then tapered over 2-3 weeks beginning on day 45 (or 15 days after engraftment if engraftment occurred \> day 30) after engraftment if there continues to be no evidence of acute GVHD. After completion of study treatment, patients are followed up at 6 months, 1 year, and 2 years.
Source references
Source notices and limitations
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