NCT00309842 · REGISTRY CONDITION SEARCH

Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases

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: 2020-09-10

RATIONALE: Giving chemotherapy drugs, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells and prepares the patient's bone marrow for the 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 may stop this from happening. PURPOSE: This phase II trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation works in treating patients who are undergoing an umbilical cord blood transplant for hematologic cancer.

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

Number of Participants Who Were Alive at 1 Year Transplant Overall Survival

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outcome · POSTED

Number of Participants Who Died Due to Transplant

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outcome · POSTED

Number of Participants With Platelet Engraftment

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outcome · POSTED

Number of Participants With Neutrophil Engraftment

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outcome · POSTED

Number of Participants With Acute Graft-Versus-Host Disease

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outcome · POSTED

Number of Participants With Chronic Graft-Versus-Host Disease

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outcome · POSTED

Percentage Chimerism on Day 21

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outcome · POSTED

Percentage Chimerism on Day 100

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outcome · POSTED

Percentage Chimerism at 6 Months

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outcome · POSTED

Percentage Chimerism at 1 Year

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outcome · POSTED

Percentage Chimerism at 2 Years

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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: * Acute myeloid leukemia (AML): high risk CR1 (as evidenced by preceding myelodysplastic syndrome \[MDS\], high risk cytogenetics, ≥ 2 cycles to obtain complete remission \[CR\], erythroblastic or megakaryocytic leukemia; CR2+. All patients must be in CR as defined by hematological recovery, AND \<5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%. * Very high risk pediatric patients with AML. Patients \<21 years, however, are eligible with (M2 marrow) with \< or = 25% blasts in marrow after having failed one or more cycles of chemotherapy. This group of patients will be analyzed separately. * Acute lymphocytic leukemia (ALL): high risk CR1 \[t(9;22), t (1:19), t(4;11) or other MLL rearrangements\] hypodiploidy, or IKZF1 abnormalities), DNA index \< 0.81, \> 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND \<5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%. * Very high risk pediatric patients with ALL. patients \<21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission * Chronic myelogenous leukemia (CML) excluding refractory blast crisis. To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate. * Plasma Cell leukemia after initial therapy, who achieved at least a partial remission * Advanced myelofibrosis * Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be \< 10% by a representative bone marrow aspirate morphology. * Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma or follicular lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting \> 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant. * Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy in CR1+ or PR1+. * Large cell NHL \> CR2/\> PR2. Patients in CR2/PR2 with initial short remission (\<6 months) are eligible. * Lymphoblastic lymphoma, Burkitt's lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II \< 1 year. * Multiple myeloma beyond PR2. Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin \> 3 mg/L, may be considered for this protocol after initial therapy. * Recipients must have a Karnofsky score (adults) ≥ 80 % or Lansky score ≥ 50% (pediatrics), and proper organ function. Exclusion Criteria * Active infection at time of transplantation * History of human immunodeficiency virus (HIV) infection * Pregnant or breast feeding. * Chemotherapy refractory large cell and high grade NHL * If \< or = 18 years old, prior myeloablative transplant within the last 6 months. If \>18 years old prior myeloablative allotransplant or autologous transplant * Extensive prior therapy including \> 12 months alkylator therapy or \> 6 months alkylator therapy with extensive radiation. * Patients who have received Y-90 ibritumomab (Zevalin) or I-131 tostumomab (Bexxar), as part of their salvage therapy.
healthy Volunteers: false
maximum Age: 55 Years
sex: ALL
std Ages: CHILD
ADULT

Treatment arms and interventions

arm Groups

description
Patients undergoing unrelated umbilical cord blood transplantation (UCBT) for hematologic malignancies treated with myeloablative preparative regimen comprising fludarabine phosphate, mycophenolate mofetil, filgrastim, cyclophosphamide, cyclosporine and fractionated total-body irradiation.
intervention Names
Biological: filgrastim
Drug: cyclophosphamide
Drug: cyclosporine
Drug: fludarabine phosphate
Drug: mycophenolate mofetil
Procedure: umbilical cord blood transplantation
Radiation: total-body irradiation
label
Unrelated UCBT for Blood Cancers
type
EXPERIMENTAL

interventions

arm Group Labels
Unrelated UCBT for Blood Cancers
description
All patients will receive G-CSF 5 mcg/kg/day intravenously(IV) (dose rounded to vial size) based on the actual body weight IV beginning on day +1 after umbilical cord blood (UCB) infusion. G-CSF will be administered daily until the absolute neutrophil count (ANC) exceeds 2.5 x 10\^9/L for three consecutive days.
name
filgrastim
other Names
G-CSF
type
BIOLOGICAL
arm Group Labels
Unrelated UCBT for Blood Cancers
description
Cyclophosphamide to be administered with high volume fluid flush and mesna (MT(S) 9006) at 10:00am, or per institutional routine, on days-7 and -6 after fludarabine. Cyclophosphamide 60mg/kg/day intravenous (IV) x 2 days, total dose 120 mg/kg (days -7 and -6) Dosing is calculated based on Actual BodyWeight (ABW) unless ABW \> 30 kg above Ideal BodyWeight (IBW), in which case the dose should be computed using adjusted body weight.
name
cyclophosphamide
other Names
Cytoxan
type
DRUG
arm Group Labels
Unrelated UCBT for Blood Cancers
description
Patients will receive cyclosporine (CSA) therapy beginning on day -3 maintaining a level of \> 200 ng/mL. For adults the initial dose will be 2.5 mg/kg intravenously (IV) over 2 hours every 12 hours. For children \< 40 kg the initial dose will be 2.5 mg/kg IV over 2 hours every 8 hours.
name
cyclosporine
other Names
CSA
type
DRUG
arm Group Labels
Unrelated UCBT for Blood Cancers
description
Fludarabine 25 mg/m2/day intravenously (IV) x 3 days, total dose 75 mg/m2 (days -8 to -6);
name
fludarabine phosphate
other Names
Fludara
type
DRUG
arm Group Labels
Unrelated UCBT for Blood Cancers
description
All patients will begin mycophenolate mofetil (MMF) on day -3. Patients ≥ 40 kilograms will receive MMF at the dose of 3 grams/day divided into 2 or 3 doses (every 12 or 8 hours). Pediatric patient (\<40 kilograms) will receive MMF at the dose of 15 mg/kg three times a day.
name
mycophenolate mofetil
other Names
MMF
type
DRUG
arm Group Labels
Unrelated UCBT for Blood Cancers
description
The product is infused via intravenous (IV) drip directly into the central line without a needle, pump or filter.
name
umbilical cord blood transplantation
other Names
UCBT
type
PROCEDURE
arm Group Labels
Unrelated UCBT for Blood Cancers
description
The recommended TBI is 165 cGy given twice daily for a total dose of 1320 cGy (days -4 to -1).
name
total-body irradiation
other Names
TBI
type
RADIATION

Study design

allocation

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 213
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Number of patients alive at 1 year after transplant.
measure
Number of Participants Who Were Alive at 1 Year Transplant Overall Survival
time Frame
at 1 year

secondary Outcomes

description
Determine the incidence of transplant-related mortality at 6 months after UCBT
measure
Number of Participants Who Died Due to Transplant
time Frame
At Month 6
description
Determine the incidence of platelet engraftment (platelet recovery \>50,000/uL) at 6 months after UCBT.
measure
Number of Participants With Platelet Engraftment
time Frame
6 months
description
Determine the incidence of neutrophil engraftment at day 42 after UCBT Patients diagnosed with graft failure (failure of absolute neutrophil count ANC \> 5 x 10\^8/L of donor origin by day +42)
measure
Number of Participants With Neutrophil Engraftment
time Frame
Day 42
description
Determine the incidence of grade II-IV and grade III-IV acute graft-versus-host disease (GVHD) at day 100 after UCBT. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria following Przepiorka et al, 1995, Consensus Clinical Stage and Grade of Acute GVHD. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level.
measure
Number of Participants With Acute Graft-Versus-Host Disease
time Frame
Day 100
description
Determine the incidence of chronic GVHD at 1 year after UCBT. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level.
measure
Number of Participants With Chronic Graft-Versus-Host Disease
time Frame
Year 1
description
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28.
measure
Percentage Chimerism on Day 21
time Frame
Day 21
description
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28.
measure
Percentage Chimerism on Day 100
time Frame
Day 100
description
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28.
measure
Percentage Chimerism at 6 Months
time Frame
Month 6
description
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28.
measure
Percentage Chimerism at 1 Year
time Frame
1 Year
description
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28.
measure
Percentage Chimerism at 2 Years
time Frame
2 Years

Full study description

brief Summary: RATIONALE: Giving chemotherapy drugs, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells and prepares the patient's bone marrow for the 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 may stop this from happening. PURPOSE: This phase II trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation works in treating patients who are undergoing an umbilical cord blood transplant for hematologic cancer.
detailed Description: OBJECTIVES: Primary * Determine the 1-year survival of patients undergoing unrelated umbilical cord blood transplantation (UCBT) for hematologic malignancies treated with myeloablative preparative regimen comprising fludarabine, cyclophosphamide, and fractionated total-body irradiation. Secondary * Determine the incidence of transplant-related mortality at 6 months after UCBT. * Evaluate the pattern of chimerism after double UCBT. * Determine the incidence of neutrophil engraftment at day 42 after UCBT. * Determine the incidence of platelet engraftment at 6 months after UCBT. * Determine the incidence of grade II-IV and grade III-IV acute graft-versus-host disease (GVHD) at day 100 after UCBT. * Determine the incidence of chronic GVHD at 1 year after UCBT. * Determine the disease-free survival at 1 and 2 years after UCBT. * Determine the incidence of relapse at 1 year after UCBT. OUTLINE: This is a nonrandomized, open-label, multicenter study. * Preparative Regimen: Patients receive fludarabine IV over 1 hour on days -8 to -6 and cyclophosphamide IV on days -7 and -6. Patients also undergo total-body irradiation twice daily on days -4 to -1. * Umbilical Cord Blood Transplantation (UCBT): Patients undergo 1 or 2 units of UCBT on day 0. Patients receive filgrastim (G-CSF) IV once daily beginning on day 1 and continuing until blood counts recover. * Graft-versus-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV over 2 hours 2 or 3 times daily beginning on day -3 and continuing until day 100 followed by a taper until day 180. Patients also receive mycophenolate mofetil IV or orally 2 or 3 times a day beginning on day -3 and continuing until day 30 or 7 days after engraftment in the absence of acute GVHD. After completion of study treatment, patients are followed periodically for at least 5 years. PROJECTED ACCRUAL: A total of 150 patients will be accrued for this study.

Source references

references

citation
Verneris MR, Brunstein CG, Barker J, MacMillan ML, DeFor T, McKenna DH, Burke MJ, Blazar BR, Miller JS, McGlave PB, Weisdorf DJ, Wagner JE. Relapse risk after umbilical cord blood transplantation: enhanced graft-versus-leukemia effect in recipients of 2 units. Blood. 2009 Nov 5;114(19):4293-9. doi: 10.1182/blood-2009-05-220525. Epub 2009 Aug 25.
pmid
19706886
type
RESULT
citation
MacMillan ML, Weisdorf DJ, Brunstein CG, Cao Q, DeFor TE, Verneris MR, Blazar BR, Wagner JE. Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. Blood. 2009 Mar 12;113(11):2410-5. doi: 10.1182/blood-2008-07-163238. Epub 2008 Nov 7.
pmid
18997171
type
DERIVED

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice