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Donor Umbilical Cord Blood Transplant in Treating Patients With Advanced Hematological Cancer or Other Disease

Phase 2
Terminated
Conditions
Leukemia
Lymphoma
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Interventions
Biological: anti-thymocyte globulin
Procedure: umbilical cord blood transplantation
Radiation: total body irradiation
Registration Number
NCT00719849
Lead Sponsor
Fred Hutchinson Cancer Center
Brief Summary

RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well donor umbilical cord blood transplant with reduced intensity conditioning works in treating patients with advanced hematological cancer or other disease.

Detailed Description

OBJECTIVES:

Primary

* To estimate the probability of survival at 1 year in patients with advanced hematological malignancies or other diseases treated with non-myeloablative unrelated donor umbilical cord blood transplantation.

Secondary

* Six month non-relapse mortality.

* Chimerism at days 7, 14, 21, 28, 56, and 80, at 6 months, and at 1 and 2 years.

* To determine the incidence of neutrophil engraftment at day 42.

* To determine the incidence of platelet engraftment at 6 months.

* To determine the incidence of grade II-IV and III-IV acute graft-versus-host-disease (GVHD) at day 100.

* To determine the incidence of chronic GVHD at 1 year.

* To determine the incidence of clinically significant infections at 6 months and at 1 and 2 years.

* To determine the probability of progression-free survival at 1 and 2 years.

* To determine the probability of survival at 2 years.

* To determine the incidence of relapse or disease progression at 1 and 2 years.

OUTLINE: Patients are stratified according to disease status and prior therapy (hematologic malignancy or other disease that was treated with an autologous stem cell transplant or ≥ 2 courses of multiagent chemotherapy within the past 3 months vs hematologic malignancy or other disease that was treated with an autologous stem cell transplant \> 12 months ago or with ≤ 1 course of multiagent chemotherapy or immunosuppressive chemotherapy within the past 3 months vs refractory leukemia or lymphoma for which patient was rendered aplastic either by induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy).

* Conditioning regimen: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV on day -6. Patients also undergo total body irradiation on day -1. Some patients also receive anti-thymocyte globulin IV on days -6 to -4.

* Umbilical cord blood transplantation (UCBT): Patients undergo UCBT on day 0.

* Immunosuppressive therapy (graft-versus-host disease prophylaxis): Patients receive cyclosporine IV over 1 hour or orally (as tolerated) every 8 or 12 hours beginning on day -3 and continuing for approximately 6 months. Patients also receive mycophenolate mofetil IV every 8 hours on days -3 to 5 and then orally on days 6-30.

After completion of study treatment, patients are followed at 6 months and then annually thereafter.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
13
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Cyclophosphamide/Fludarabine/TBIumbilical cord blood transplantationSubjects with hematological malignancies with prior autologous transplant, \>2 cycles of multiagent chemotherapy, or severely immune suppressive therapy in last 3 months. Refractory leukemia and lymphoma in aplasia after induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy.
Cyclophosphamide/Fludarabine/TBItotal body irradiationSubjects with hematological malignancies with prior autologous transplant, \>2 cycles of multiagent chemotherapy, or severely immune suppressive therapy in last 3 months. Refractory leukemia and lymphoma in aplasia after induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy.
Cyclophosphamide/Fludarabine/TBI/ATGanti-thymocyte globulinSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Cyclophosphamide/Fludarabine/TBI/ATGfludarabine phosphateSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Cyclophosphamide/Fludarabine/TBI/ATGumbilical cord blood transplantationSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Cyclophosphamide/Fludarabine/TBI/ATGtotal body irradiationSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Cyclophosphamide/Fludarabine/TBIcyclosporineSubjects with hematological malignancies with prior autologous transplant, \>2 cycles of multiagent chemotherapy, or severely immune suppressive therapy in last 3 months. Refractory leukemia and lymphoma in aplasia after induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy.
Cyclophosphamide/Fludarabine/TBIcyclophosphamideSubjects with hematological malignancies with prior autologous transplant, \>2 cycles of multiagent chemotherapy, or severely immune suppressive therapy in last 3 months. Refractory leukemia and lymphoma in aplasia after induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy.
Cyclophosphamide/Fludarabine/TBIfludarabine phosphateSubjects with hematological malignancies with prior autologous transplant, \>2 cycles of multiagent chemotherapy, or severely immune suppressive therapy in last 3 months. Refractory leukemia and lymphoma in aplasia after induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy.
Cyclophosphamide/Fludarabine/TBImycophenolate mofetilSubjects with hematological malignancies with prior autologous transplant, \>2 cycles of multiagent chemotherapy, or severely immune suppressive therapy in last 3 months. Refractory leukemia and lymphoma in aplasia after induction chemotherapy or radioimmunoconjugated monoclonal antibody therapy.
Cyclophosphamide/Fludarabine/TBI/ATGcyclophosphamideSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Cyclophosphamide/Fludarabine/TBI/ATGcyclosporineSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Cyclophosphamide/Fludarabine/TBI/ATGmycophenolate mofetilSubjects with hematological malignancies with prior autologous transplant \>12 mos or \<1 cycle of multiagent chemotherapy or NO immune suppressive chemotherapy in last 3 months
Primary Outcome Measures
NameTimeMethod
Probability of Survival at 1 Year1 year post transplant

Kaplan-Meier estimate of the probability of survival at 1 year

Secondary Outcome Measures
NameTimeMethod
Probability of Survival at 2 Years2 years post transplant

Kaplan-Meier estimate of the probability of survival at 2 years

Incidence of Non-relapse Mortality at 6 Months6 months post transplant

Number of Participants with Non-relapse Mortality at 6 Months

Chimerism7 days, 14 days, 21 days, 28 days, 56 days, and 80 days, 6 months, 1 and 2 years post transplant

Count of participants who experienced dominance of one cord blood unit (defined by \>or= 95% contribution of one cord blood unit to BM and all PB fractions -- CD3+, CD33+, CD56+, and CD19+) at 7 days, 14 days, 21 days, 28 days, 56 days, and 80 days, 6 months, 1 and 2 years post transplant.

Incidence of Neutrophil Engraftment at Day 42Day 42 post transplant

Number of participants with neutrophil engraftment at day 42

Incidence of Platelet Engraftment at 6 Months6 months post transplant

Number of participants with platelet engraftment at 6 months

Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD) at Day 100Day 100 post transplant

Number of participants with Grade II-IV acute graft-versus-host-disease (GVHD) at day 100.

Acute GVHD Staging and Grading:

Overall grade 1: stage 1-2 skin, no liver or gut Overall grade 2: stage 3 skin or stage 1 liver or stage 1 gut Overall grade 3: stage 4 skin or stage 2-4 liver or stage 2-4 gut (without GVHD as a major contributing cause of death) Overall grade 4: stage 4 skin or stage 2-4 liver or stage 2-3 gut (with GVHD as a major contributing cause of death)

Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD) at Day 100Day 100 post transplant

Number of participants with Grade III-IV acute graft-versus-host-disease (GVHD) at day 100.

Acute GVHD Staging and Grading:

Overall grade 1: stage 1-2 skin, no liver or gut Overall grade 2: stage 3 skin or stage 1 liver or stage 1 gut Overall grade 3: stage 4 skin or stage 2-4 liver or stage 2-4 gut (without GVHD as a major contributing cause of death) Overall grade 4: stage 4 skin or stage 2-4 liver or stage 2-3 gut (with GVHD as a major contributing cause of death)

Incidence of Chronic Graft-versus-host-disease (GVHD) at 1 Year1 year post transplant

Number of participants with chronic graft-versus-host-disease (GVHD) at 1 year.

Clinical Limited cGVHD

1. Oral abnormalities consistent with cGVHD, a positive skin or lip biopsy, and no other manifestations of cGVHD.

2. Mild liver test abnormalities (alkaline phosphatase \<2 x upper limit of normal, AST or ALT \<3 x upper limit of normal and total bilirubin \<1.6) with positive skin or lip biopsy, and no other manifestations of cGVHD.

3. Less than 6 papulosquamous plaques, macular-papular or lichenoid rash involving \<20% of body surface area (BSA), dyspigmentation involving \<20% BSA, or erythema involving \<50% BSA, positive skin biopsy, and no other manifestations of cGVHD.

4. Ocular sicca (Schirmer's test \<5mm with no more than minimal ocular symptoms), positive skin or lip biopsy, and no other manifestations of cGVHD.

5. Vaginal or vulvar abnormalities with positive biopsy, and no other manifestations of cGVHD.

Incidence of Clinically Significant Infections at 6 Months6 months post transplant

Number of participants with clinically significant infections at 6 months

Incidence of Clinically Significant Infections at 1 Year1 year post transplant

Number of participants with clinically significant infections at 1 year

Incidence of Clinically Significant Infections at 2 Years2 years post transplant

Number of participants with clinically significant infections at 2 years

Probability of Progression-free Survival at 1 Year1 year post transplant

Kaplan-Meier estimate of the probability of progression-free survival at 1 year

Probability of Progression-free Survival at 2 Years2 years post transplant

Kaplan-Meier estimate of the probability of progression-free survival at 2 years

Incidence of Relapse at 1 Year1 year post transplant

Number of participants with relapse at 1 year.

Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Incidence of Relapse at 2 Years2 years post transplant

Number of participants with relapse at 2 years.

Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Trial Locations

Locations (1)

Fred Hutchinson Cancer Research Center

🇺🇸

Seattle, Washington, United States

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