NCT01093586 · REGISTRY CONDITION SEARCH

Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

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: 2019-01-23

RATIONALE: Giving chemotherapy before a donor umbilical cord blood transplant (UCBT) 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 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. Giving antithymocyte globulin before transplant and cyclosporine and mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well donor umbilical cord blood stem cell transplant works in treating patients with hematologic malignancies.

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

Hematologic Engraftment

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outcome · POSTED

Overall Survival

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outcome · POSTED

Overall Survival

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outcome · POSTED

Disease Free

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outcome · POSTED

Disease Free

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outcome · POSTED

Recurrence or Relapse

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outcome · POSTED

Recurrence or Relapse

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outcome · POSTED

Transplant Related Mortality

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outcome · POSTED

Transplant Related Mortality

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outcome · POSTED

Occurrence of Serious Infections

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outcome · POSTED

Immune Reconstitution

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outcome · POSTED

Toxicity Related to UCB Transplantation and Cytoreduction as Assessed by CTC v3.0

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outcome · POSTED

Incidence of Acute Graft-versus-host Disease (GVHD)

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outcome · POSTED

Incidence of Chronic GVHD

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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: * Patients will be diagnosed with one of the following hematological malignancies: acute myelogenous leukemia (AML), acute lymphoblastic leukemia, non-Hodgkin's lymphoma, myelodysplastic syndrome (MDS), chronic myelogenous leukemia (CML), and myeloproliferative and lymphoproliferative disorders * AML--First remission (CR1) with high risk features including a known prior diagnosis of myelodysplasia (MDS); therapy related AML; white cell count at presentation \> 100,000; presence of extramedullary leukemia at diagnosis; unfavorable AML subtype (M0, M5-M7); poor cytogenetic markers (abnormalities of chromosome 5, 7 or 8, 11q23, Philadelphia chromosome, complex karyotype) * AML--Second remission (CR2) or subsequent remission * AML--Relapse/Persistent Disease with \< 20% bone marrow blasts * ALL--First remission (CR1) at high risk for relapse as defined by: B cell ALL white blood cell count (WBC) at presentation \> 30,000 (T cell ALL WBC \> 100,000); presence of high-risk cytogenetic abnormality such as t(9;22), t(1;19), t(4;11) or other MLL rearrangements (11q23), t(8;14) * ALL--Second remission (CR2) or subsequent remission * ALL--Relapse/Persistent Disease with \< 20% bone marrow blasts * Non-Hodgkin Lymphoma--Induction failure or relapse and sensitive to most recent chemotherapy * MDS--Low or Intermediate-1 International Prognostic Scoring System (IPSS) score with: life-threatening cytopenia(s); and/or red cell or platelet transfusion dependence * MDS--ANC \< 500, recurrent infections, PRBC transfusions \> 2 units/month, poor risk cytogenetics, platelet transfusion dependence * MDS--Intermediate-2 or High IPSS score * CML--Chronic phase I (CP1) and resistant to or intolerant of tyrosine kinase inhibitors (i.e. imatinib, dasatinib, etc.) * CML--CP2 or subsequent chronic phase, including chronic phase achieved after induction therapy for blast crisis * Myeloproliferative and lymphoproliferative disorders--eligibility to be determined by a consensus of the physicians on the Case Comprehensive Cancer Center Leukemia/Lymphoma Multidisciplinary Committee * Myeloproliferative and lymphoproliferative disorders--must have evidence of disease acceleration to be a candidate for umbilical cord blood transplant; myeloproliferative disorders eligible for transplant include chronic myelomonocytic leukemia (CMML) with high IPSS score and myelofibrosis * Myeloproliferative and lymphoproliferative disorders--potential lymphoproliferative disorders eligible for transplant include chronic lymphocytic leukemia, prolymphocytic leukemia, and large granular lymphocytic leukemia * Good performance status: Karnofsky \>= 70 % or ECOG 0-1 * Calculated creatinine clearance \>= 60 mL/min, or measured creatinine clearance \>= 60 mL/min (by 24-hour urine collection) if creatinine \>= 1.5 or history of renal dysfunction * Hepatic Transaminases \< 4 x upper limit normal (ULN); total bilirubin \< 2.5 mg/dL, unless the patient has a history of benign congenital hyperbilirubinemia (Gilbert's syndrome) * Normal cardiac function by echocardiogram or radionuclide scan, (left ventricular ejection fraction \> 45%); if the left ventricular ejection fraction is between 40-50%, clearance by an adult cardiologist is required * Pulmonary function tests demonstrating FEV1 \> 60% of predicted for age * Adults must have a DLCOva \> 60% normal * For patients unable to complete pulmonary function tests clearance by an adult pulmonologist is required * Patients will be eligible for the clinical trial under the following conditions: they do NOT have an HLA-A/B/DR B1 identical RELATED bone marrow donor; they do NOT have a 6/6 HLA-identical matched unrelated adult donor; OR a matched related donor transplant is not in the best interest of the patient (i.e., patient's condition precludes waiting on the donor, too much time to prepare the donor, the donor is ineligible due to medical reasons, or in the case of high risk disease a related donor is not appropriated (syngeneic transplant); the decision must be agreed upon by the consensus of physicians on the Case Comprehensive Cancer Center Leukemia/Lymphoma Multidisciplinary Committee; OR their condition precludes waiting to search and find a donor in the National Marrow Donor Registry Exclusion Criteria: * Female patients who are pregnant or breast-feeding * HIV or HTLV-1 positivity * Any leukemia with a morphologic relapse or persistent disease in the BM with \>= 20% blasts (cytogenetic relapse without morphologic evidence of relapse, or cytogenetic persistent disease is acceptable) * Active extramedullary leukemia, including CNS disease * Prior hematopoietic stem cell transplant (autologous or allogeneic) * Uncontrolled infection * Patient has an identical related bone marrow donor or a 6/6 HLA-identical matched unrelated donor * Any patient who is unable to provide informed consent or comply with the requirements of the protocol
healthy Volunteers: false
maximum Age: 64 Years
minimum Age: 12 Years
sex: ALL
std Ages: CHILD
ADULT

Treatment arms and interventions

arm Groups

description
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
intervention Names
Procedure: double-unit umbilical cord blood transplantation
Other: cytogenetic analysis
Procedure: bone marrow aspiration
Other: fluorescence in situ hybridization
Drug: busulfan
Drug: cyclophosphamide
Drug: anti-thymocyte globulin
Drug: methylprednisolone
Drug: cyclosporine
Drug: mycophenolate mofetil
Other: flow cytometry
Procedure: allogeneic hematopoietic stem cell transplantation
label
Arm I
type
EXPERIMENTAL

interventions

arm Group Labels
Arm I
description
Undergo transplantation
name
double-unit umbilical cord blood transplantation
type
PROCEDURE
arm Group Labels
Arm I
description
Correlative studies
name
cytogenetic analysis
type
OTHER
arm Group Labels
Arm I
description
Correlative studies
name
bone marrow aspiration
type
PROCEDURE
arm Group Labels
Arm I
description
Correlative studies
name
fluorescence in situ hybridization
other Names
fluorescence in situ hybridization (FISH)
type
OTHER
arm Group Labels
Arm I
description
Given orally
name
busulfan
other Names
BSF
BU
Misulfan
Mitosan
Myeloleukon
Myelosan
type
DRUG
arm Group Labels
Arm I
description
Given IV
name
cyclophosphamide
other Names
CPM
CTX
Cytoxan
Endoxan
Endoxana
Enduxan
type
DRUG
arm Group Labels
Arm I
description
Given IV
name
anti-thymocyte globulin
other Names
ATG
ATGAM
lymphocyte immune globulin
Thymoglobulin
type
DRUG
arm Group Labels
Arm I
description
Given IV
name
methylprednisolone
other Names
A-MethaPred
Depo-Medrol
Medrol
MePRDL
Solu-Medrol
Wyacort
type
DRUG
arm Group Labels
Arm I
description
Given IV
name
cyclosporine
other Names
27-400
ciclosporin
cyclosporin
cyclosporin A
CYSP
Sandimmune
type
DRUG
arm Group Labels
Arm I
description
Given orally or IV
name
mycophenolate mofetil
other Names
Cellcept
MMF
type
DRUG
arm Group Labels
Arm I
description
Correlative studies
name
flow cytometry
type
OTHER
arm Group Labels
Arm I
description
Undergo transplantation
name
allogeneic hematopoietic stem cell transplantation
type
PROCEDURE

Study design

allocation

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 14
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Number of participants alive at 180 days post engraftment.
measure
Overall Survival
time Frame
On day +180

secondary Outcomes

description
Number of participants that were able to complete engraftment by day 42.
measure
Hematologic Engraftment
time Frame
On day +42
description
Number of participants that were alive.
measure
Overall Survival
time Frame
At 1 year
description
Number of participants that were alive.
measure
Overall Survival
time Frame
At 2 years
description
Number of participants that were disease free
measure
Disease Free
time Frame
At 1 year
description
Number of participants that were disease free
measure
Disease Free
time Frame
At 2 years
description
Number of subjects that had disease recurrence
measure
Recurrence or Relapse
time Frame
one year in patients post UCBT
description
Number of subjects that had disease recurrence
measure
Recurrence or Relapse
time Frame
two years post transplant
description
Number of subjects that died because of transplant
measure
Transplant Related Mortality
time Frame
On day 100 post transplant
description
Number of subjects that died because of transplant
measure
Transplant Related Mortality
time Frame
On day 180 post transplant
description
Number of participants that had infections
measure
Occurrence of Serious Infections
time Frame
1 year
description
Immunodificency panel to see recovery of immune system. Number of participants that recovered.
measure
Immune Reconstitution
time Frame
Periodically for 2 years
description
Number of participants that experienced toxicity related to the transplant
measure
Toxicity Related to UCB Transplantation and Cytoreduction as Assessed by CTC v3.0
time Frame
by day +42

Full study description

brief Summary: RATIONALE: Giving chemotherapy before a donor umbilical cord blood transplant (UCBT) 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 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. Giving antithymocyte globulin before transplant and cyclosporine and mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well donor umbilical cord blood stem cell transplant works in treating patients with hematologic malignancies.
detailed Description: PRIMARY OBJECTIVES: 1\. To establish the day +180 overall survival after a myeloablative unrelated double unit UCBT in a single institution setting. SECONDARY OBJECTIVES: 1. To determine the rates of hematologic and immune reconstitution in patients with high risk hematologic malignancies, who are undergoing myeloablative chemotherapy followed by infusion of double unit UCBT. 2. To determine the contribution of each umbilical cord unit to immune reconstitution with a focus on both initial (day +21 BM, and +28 PB) and sustained engraftment (day +100 BM; PB at +14, +21, +28, +35, +42, +60, +100, +180, +1 and 2 years). 3. To determine the probability of overall survival and disease free survival at one and two years. 4. To describe the incidence of disease recurrence at one and two years in patients post UCBT. 5. To describe the incidence of acute GVHD and chronic GVHD at 100 days and at one year, respectively. 6. To determine the incidence of day 100 and 180 treatment related mortality. 7. To determine the incidence of serious infectious complications in the first year after transplant. 8. To determine the incidence of donor-derived neutrophil and platelet recovery. 9. To determine the incidence of secondary lymphoproliferative diseases following transplantation with umbilical cord blood. OUTLINE: PREPARATIVE REGIMEN: Patients receive oral busulfan every 6 hours on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally every 8 hours on days -3 to 45. After completion of study treatment, patients are followed periodically.

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice