Donor Umbilical Cord Blood Transplant After Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Cancer
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Purpose
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer 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).
PURPOSE: This phase I trial is studying the safety of donor umbilical cord blood transplant after fludarabine phosphate, cyclophosphamide, and total-body irradiation in treating patients with high-risk hematologic cancer (now closed).
The Phase II part of this trial is studying whether priming one of two UCB units with C3a facilitates engraftment of the treated unit.
| Condition | Intervention | Phase |
|---|---|---|
|
Leukemia Lymphoma Multiple Myeloma Myelodysplastic Syndromes |
Drug: cyclophosphamide Drug: fludarabine phosphate Radiation: Total body irradiation Biological: Umbilical cord blood unit with C3a fragment Biological: Unmanipulated UCB Unit |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | MT2008-15: Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Nonmyeloablative Preparative Regimen and Priming With Complement 3a Fragment (C3a) |
- Proportion of patients with the complement 3a (C3a) unit predominating [ Time Frame: Day 180 ] [ Designated as safety issue: No ]Efficacy of the pre-incubation of one of two umbilical cord blood (UCB) units with C3a as part of a unrelated donor double UCB nonmyeloablative transplantation in patients with high-risk hematological malignancies.
- Neutrophil Engraftment [ Time Frame: Day 42 ] [ Designated as safety issue: No ]Achieving 500 neutrophils/uL by day 42.
- Blood Chimerism [ Time Frame: Days 7, 14, 21, 28, 60 and 100 ] [ Designated as safety issue: No ]Proportion of donor DNA present in the peripheral blood
- Incidence of grades II-IV and III-IV graft-vs-host disease [ Time Frame: Day 0 through Day 100 ] [ Designated as safety issue: Yes ]Development of graft-versus-host disease
- Non-Relapse Mortality [ Time Frame: Day 180 and 360 ] [ Designated as safety issue: No ]Deaths not due to relapse.
- Overall survival [ Time Frame: Day 360 and 720 ] [ Designated as safety issue: No ]Survival (alive) from transplantation to last follow-up.
- Bone Marrow Chimerism [ Time Frame: Day 21, 100, 180, 360 and 720 ] [ Designated as safety issue: No ]Proportion of donor DNA in the bone marrow.
- Chronic Graft-Versus-Host Disease [ Time Frame: Day 360 ] [ Designated as safety issue: No ]Patients who developed chronic graft-versus-host disease.
- Relapse of Disease [ Time Frame: Day 360 and 720 ] [ Designated as safety issue: No ]Patients who developed disease relapse after transplantation.
- Disease Progression [ Time Frame: Day 360 and 720 ] [ Designated as safety issue: No ]Patients who developed disease progression after transplantation.
- Platelet Recovery [ Time Frame: Day 180 ] [ Designated as safety issue: No ]Proportion of patients with >50,000 or >20,000 platelets/uL by day 180
| Enrollment: | 34 |
| Study Start Date: | March 2010 |
| Estimated Study Completion Date: | August 2014 |
| Primary Completion Date: | May 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Complement Fragment 3A - Small Cell Dose
Patients who received complement fragment 3a (C3a) priming of the UCB unit with the smaller cell dose following preparation regimen and radiation.
|
Drug: cyclophosphamide
50 mg/kg intravenously (IV) over 2 hours on Day -6.
Other Name: Cytoxan
Drug: fludarabine phosphate
40 mg/m^2 over 1 hour on Days -6 through -2.
Other Name: Fludara
Radiation: Total body irradiation
200 cGy on Day -1
Biological: Umbilical cord blood unit with C3a fragment
On Day 0, the C3a primed UCB unit will be infused intravenously SECOND, within 30 minutes of the completion of the infusion of the unmanipulated UCB unit, through a central line without in-line filtration in a manner identical to the unmanipulated UCB unit.
Biological: Unmanipulated UCB Unit
On Day 0, the unmanipulated UCB unit will be infused FIRST through a central line without in-line filtration per institutional guidelines.
|
|
Experimental: Complement Fragment A - Larger Cell Dose
Patients who received complement fragment 3a (C3a) priming of the UCB unit with the larger cell dose.
|
Drug: cyclophosphamide
50 mg/kg intravenously (IV) over 2 hours on Day -6.
Other Name: Cytoxan
Drug: fludarabine phosphate
40 mg/m^2 over 1 hour on Days -6 through -2.
Other Name: Fludara
Radiation: Total body irradiation
200 cGy on Day -1
Biological: Umbilical cord blood unit with C3a fragment
On Day 0, the C3a primed UCB unit will be infused intravenously SECOND, within 30 minutes of the completion of the infusion of the unmanipulated UCB unit, through a central line without in-line filtration in a manner identical to the unmanipulated UCB unit.
Biological: Unmanipulated UCB Unit
On Day 0, the unmanipulated UCB unit will be infused FIRST through a central line without in-line filtration per institutional guidelines.
|
Detailed Description:
OUTLINE:
- Nonmyeloablative preparative regimen: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 2 hours on day -6. Patients then undergo total-body irradiation on day -1. Some patients also receive anti-thymocyte globulin IV every 12 hours on days -6, -5, and -4.
- Umbilical cord blood (UCB) transplantation: Patients undergo unmanipulated UCB transplantation followed by complement 3a fragment primed UCB transplantation on day 0.
Treatment for graft-vs-host disease prophylaxis is also given.
After completion of study therapy, patients are followed up periodically for up to 2 years.
Eligibility| Ages Eligible for Study: | 18 Years to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Disease Criteria
- Acute Leukemias: Must be in remission by morphology (<5% blasts). Note cytogenetic relapse or persistent disease without morphologic relapse is acceptable. Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse. (See exclusion criteria for more detailed definition)
- Acute myeloid leukemia: high risk CR1 (as evidenced by preceding myelodysplastic syndrome (MDS), high risk cytogenetics such as those associated with MDS or complex karyotype, > 2 cycles to obtain complete remission (CR) or erythroblastic and megakaryocytic); second or greater CR.
- Acute lymphoblastic leukemia/lymphoma: high risk CR1 as evidenced by high risk cytogenetics (e.g. t(9;22, t(1;19), t(4;11), other MLL rearrangements) or > 1 cycle to obtain CR; second or greater CR.
- Burkitt's lymphoma in CR2 or subsequent CR
- Natural Killer cell malignancies
- Myeloproliferative syndromes/diseases: Chronic myelogenous leukemia (CML) in chronic or accelerated phase but patients must have failed or been intolerant to Imatinib mesylate. EXCLUDED: CML in refractory blast crisis, myelofibrosis, polycythemia vera, and essential thrombocytosis.
- Myelodysplastic Syndrome: any subtype including refractory anemia (RA) if severe pancytopenia, high risk complex cytogenetics or International Prognostic Scoring System (IPSS) ≥ intermediate-2 (Int-2). Blasts must be less than 5%. If 5% or more requires therapy pre-transplant to reduce blast count to ≤5%. Patients who receive single agent 5-azacytidine, decitabine or immunomodulating drugs are eligible.
- Large-cell lymphoma, Hodgkin's lymphoma and multiple myeloma: patients with chemotherapy sensitive disease that has failed or who are ineligible for an autologous transplant. Patients are eligible for umbilical cord blood (UCB) transplantation if there is no evidence of progressive disease by imaging modalities and/or biopsy. Persistent PET activity, though possibly related to lymphoma, IS NOT an exclusion criterion in the absence of computated tomography (CT) changes in size indicating progression. Large-cell and Hodgkin's lymphoma that is progressive on salvage therapy is NOT eligible. Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma which have progressed after at least two prior therapies. Patients with bulky disease should be considered for debulking chemotherapy before transplant. Patients with refractory disease are eligible, unless has bulky disease and an estimated tumor doubling time of less than one month. Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy if chemotherapy sensitive. Mantle-cell lymphoma that is progressive on salvage therapy is NOT eligible. Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Umbilical Cord Blood Graft Selection - Two UCB units will be compose the graft, and each unit must be a 4-6 HLA-A, B, DRB1 antigen match to each other, as well as a 4-6 antigen match to the recipient. The combined cryopreserved nucleated cell dose of the 2 units must be ≥ 3 X 10^7/kg with each unit having a minimum cell dose of 1.5 X 10^7/kg. UCB units will be selected according to a common umbilical cord blood graft selection algorithm
- Performance Status - adequate performance status defined as Karnofsky score ≥ 60
- Age 18 to 70 years of age; patients ≥ 70 but ≤ 75 years are eligible if the co-morbidity score is ≤ 2
Organ Function
- Cardiac: Left ventricular ejection fraction > 35%; absence of decompensated congestive heart failure; absence of uncontrolled arrhythmia
- Pulmonary: DLCO > 30% of predicted; absence of O2 requirements
- Hepatic: ALT, AST, alkaline phosphatase and bilirubin < 5 x upper limit of normal
- Renal: Creatinine ≤ 2 mg/dl (patients with a creatinine > 1.2 or history of renal dysfunction must have calculated glomerular filtration rate (GFR) > 40 mL/min/1.73m2)
- If recent mold infection e.g. Aspergillus - must have minimum of 30 days of appropriate treatment before transplant and infection controlled and be cleared by Infectious Disease.
The following conditions must be met:
- If prior myeloablative autologous transplant, must be > 3 months but ≤ 12 months from transplant OR have received at least 2 cycles of multi-agent or highly immunosuppressive chemotherapy (i.e. induction for acute leukemia) within the 3 months preceding this study. OR
- If neither prior myeloablative autologous transplant ≤ 12 months from transplant nor have received at least 2 cycles of multi-agent or highly immunosuppressive chemotherapy (i.e. induction for acute leukemia) within the 3 months preceding this study, patients are eligible as long as they receive equine anti-thymocyte globulin as part of the conditioning regimen.
Exclusion Criteria:
- Patients who have an available, medically suitable, 5-6/6 HLA-A, B, DRB1 matched sibling donor
- Patients who are eligible for autologous transplantation
- Prior allogeneic transplant
- Acquired or inherited bone marrow failure syndromes such as aplastic anemia and Fanconi anemia
- Pregnant or breast feeding
- Evidence of HIV infection or known HIV positive serology
- Current uncontrolled serious infection
- Active central nervous system malignancy
Contacts and Locations| United States, Minnesota | |
| University of Minnesota Medical Center - Fairview | |
| Minneapolis, Minnesota, United States, 55455 | |
| Principal Investigator: | Claudio G. Brunstein, MD, PhD | Masonic Cancer Center, University of Minnesota |
More Information
Additional Information:
No publications provided
| Responsible Party: | Masonic Cancer Center, University of Minnesota |
| ClinicalTrials.gov Identifier: | NCT00963872 History of Changes |
| Other Study ID Numbers: | 2008LS097, MT2008-15, 0809M46361, RC1HL099447 |
| Study First Received: | August 21, 2009 |
| Last Updated: | March 15, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Leukemia Lymphoma Multiple Myeloma Neoplasms, Plasma Cell Myelodysplastic Syndromes Preleukemia Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Hemostatic Disorders Vascular Diseases Cardiovascular Diseases |
Paraproteinemias Blood Protein Disorders Hematologic Diseases Hemorrhagic Disorders Bone Marrow Diseases Precancerous Conditions Complement System Proteins Cyclophosphamide Fludarabine monophosphate Fludarabine Vidarabine Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Immunosuppressive Agents |
ClinicalTrials.gov processed this record on May 23, 2013