- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT00963872
Donor Umbilical Cord Blood Transplant After Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Cancer
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)
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.
Studieoversigt
Status
Betingelser
Detaljeret beskrivelse
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.
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 2
- Fase 1
Kontakter og lokationer
Studiesteder
-
-
Minnesota
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Minneapolis, Minnesota, Forenede Stater, 55455
- University of Minnesota Medical Center - Fairview
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
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
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: 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.
|
200 cGy på dag -1
50 mg/kg intravenously (IV) over 2 hours on Day -6.
Andre navne:
40 mg/m^2 over 1 hour on Days -6 through -2.
Andre navne:
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.
On Day 0, the unmanipulated UCB unit will be infused FIRST through a central line without in-line filtration per institutional guidelines.
|
|
Eksperimentel: Complement Fragment A - Larger Cell Dose
Patients who received complement fragment 3a (C3a) priming of the UCB unit with the larger cell dose.
|
200 cGy på dag -1
50 mg/kg intravenously (IV) over 2 hours on Day -6.
Andre navne:
40 mg/m^2 over 1 hour on Days -6 through -2.
Andre navne:
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.
On Day 0, the unmanipulated UCB unit will be infused FIRST through a central line without in-line filtration per institutional guidelines.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Number of Patients With the Complement 3a (C3a) Unit Predominating
Tidsramme: Day 180
|
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.
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Day 180
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Neutrophil Engraftment
Tidsramme: Day 42
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Achieving 500 neutrophils/uL by day 42.
|
Day 42
|
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Donor Chimerism in Blood
Tidsramme: Day 28
|
Percentage of donor DNA present in the peripheral blood
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Day 28
|
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Incidence of Grades II-IV Graft-vs-host Disease
Tidsramme: Day 0 through Day 100
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Development of graft-versus-host disease through day 100.
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Day 0 through Day 100
|
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Non-Relapse Mortality
Tidsramme: Day 180
|
Deaths not due to relapse.
|
Day 180
|
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Overall Survival
Tidsramme: Day 360
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Survival (alive) from transplantation to last follow-up.
|
Day 360
|
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Bone Marrow Chimerism
Tidsramme: Day 21
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Percentage of donor DNA in the bone marrow.
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Day 21
|
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Chronic Graft-Versus-Host Disease
Tidsramme: Day 360
|
Patients who developed chronic graft-versus-host disease.
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Day 360
|
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Relapse of Disease
Tidsramme: Day 360
|
Patients who developed disease relapse after transplantation.
|
Day 360
|
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Disease Progression
Tidsramme: Day 360
|
Patients who developed disease progression after transplantation.
|
Day 360
|
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Platelet Recovery
Tidsramme: Day 180
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Number of patients with >20,000 platelets/uL by day 180
|
Day 180
|
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Donor Chimerism in Blood
Tidsramme: Day 60
|
Percentage of donor DNA present in the peripheral blood
|
Day 60
|
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Incidence of Grades III-IV Graft-vs-host Disease
Tidsramme: 0 to 100 days
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Development of graft-versus-host disease by day 100.
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0 to 100 days
|
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Non-relapse Mortality
Tidsramme: Day 360
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Deaths not due to relapse.
|
Day 360
|
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Overall Survival at Day 720
Tidsramme: 720 days
|
Survival (alive) from transplantation to last follow-up at day 720.
|
720 days
|
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Relapse of Disease
Tidsramme: Day 720
|
Patients who developed disease relapse after transplantation.
|
Day 720
|
|
Disease Progression
Tidsramme: Day 720
|
Patients who developed disease progression after transplantation.
|
Day 720
|
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Donor Chimerism
Tidsramme: Day 100
|
Percentage of donor DNA in the bone marrow.
|
Day 100
|
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Donor Chimerism
Tidsramme: Day 180
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Percentage of donor DNA in the bone marrow.
|
Day 180
|
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Donor Chimerism
Tidsramme: Day 360
|
Percentage of donor DNA in the bone marrow.
|
Day 360
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Samarbejdspartnere og efterforskere
Samarbejdspartnere
Efterforskere
- Ledende efterforsker: Claudio G. Brunstein, MD, PhD, Masonic Cancer Center, University of Minnesota
Datoer for undersøgelser
Studer store datoer
Studiestart
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Skøn)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Hjerte-kar-sygdomme
- Karsygdomme
- Sygdomme i immunsystemet
- Neoplasmer efter histologisk type
- Neoplasmer
- Lymfoproliferative lidelser
- Immunproliferative lidelser
- Knoglemarvssygdomme
- Hæmatologiske sygdomme
- Hæmoragiske lidelser
- Hæmostatiske lidelser
- Paraproteinæmier
- Blodproteinforstyrrelser
- Neoplasmer, Plasmacelle
- Myelodysplastiske syndromer
- Myelomatose
- Lægemidlers fysiologiske virkninger
- Molekylære mekanismer for farmakologisk virkning
- Antirheumatiske midler
- Antimetabolitter, Antineoplastisk
- Antimetabolitter
- Antineoplastiske midler
- Immunsuppressive midler
- Immunologiske faktorer
- Antineoplastiske midler, Alkylering
- Alkyleringsmidler
- Myeloablative agonister
- Cyclofosfamid
- Fludarabin
- Fludarabin phosphat
Andre undersøgelses-id-numre
- 2008UC097
- MT2008-15 (Anden identifikator: Blood and Marrow Transplantation Program)
- 0809M46361 (Anden identifikator: IRB, University of Minnesota)
- RC1HL099447 (U.S. NIH-bevilling/kontrakt)
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