Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
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|ClinicalTrials.gov Identifier: NCT03333486|
Recruitment Status : Recruiting
First Posted : November 7, 2017
Last Update Posted : December 20, 2018
|Condition or disease||Intervention/treatment||Phase|
|Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive Acute Leukemia in Remission Acute Lymphoblastic Leukemia Acute Myeloid Leukemia Acute Myeloid Leukemia With FLT3/ITD Mutation Acute Myeloid Leukemia With Gene Mutations Aplastic Anemia B-Cell Non-Hodgkin Lymphoma CD40 Ligand Deficiency Chronic Granulomatous Disease Chronic Leukemia in Remission Chronic Lymphocytic Leukemia Chronic Myelogenous Leukemia, BCR-ABL1 Positive Chronic Myelomonocytic Leukemia Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive Congenital Amegakaryocytic Thrombocytopenia Congenital Neutropenia Congenital Pure Red Cell Aplasia Glanzmann Thrombasthenia Immunodeficiency Syndrome Myelodysplastic Syndrome Myelofibrosis Myeloproliferative Neoplasm Paroxysmal Nocturnal Hemoglobinuria Plasma Cell Myeloma Polycythemia Vera Recurrent Non-Hodgkin Lymphoma Refractory Non-Hodgkin Lymphoma Secondary Acute Myeloid Leukemia Secondary Myelodysplastic Syndrome Severe Aplastic Anemia Shwachman-Diamond Syndrome Sickle Cell Disease T-Cell Non-Hodgkin Lymphoma Thalassemia Waldenstrom Macroglobulinemia Wiskott-Aldrich Syndrome||Drug: Cyclophosphamide Drug: Fludarabine Phosphate Other: Laboratory Biomarker Analysis Procedure: Peripheral Blood Stem Cell Transplantation Radiation: Total-Body Irradiation||Phase 2|
I. To evaluate the rate of relapse, defined as recurrence of underlying disease or progression of underlying disease, at 1 year in patients who receive haploidentical peripheral blood stem cells (PBSCs) after reduced intensity conditioning and post-transplant cyclophosphamide.
I. To evaluate safety including development of acute graft versus host disease (GVHD) and death at 100 days post-transplant, as well as other treatment related toxicities including chronic GVHD, engraftment rate, non-relapse mortality, progression free survival (PFS) at one year, and overall survival (OS) at one year, as compared with historical controls.
I. Correlative studies will include chimerism analysis by molecular analysis and evaluation of immune reconstitution by cytomegalovirus (CMV) dextramer analysis using flow cytometry.
Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 2 hours on days -6 and -5. Patients undergo total body irradiation (TBI) on days -1 and peripheral blood stem cell transplantation (PBSCT) on day 0.
After completion of study treatment, patients are followed up at 30 and 100 days.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||58 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells|
|Actual Study Start Date :||December 7, 2017|
|Estimated Primary Completion Date :||September 6, 2021|
|Estimated Study Completion Date :||September 6, 2022|
Experimental: Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
Patients receive fludarabine phosphate IV over 30 minutes on days -6 to -2 and cyclophosphamide IV over 2 hours on days -6 and -5. Patients undergo TBI on days -1 and PBSCT on day 0.
Drug: Fludarabine Phosphate
Other: Laboratory Biomarker Analysis
Procedure: Peripheral Blood Stem Cell Transplantation
Radiation: Total-Body Irradiation
- Relapse rate [ Time Frame: At 1 year ]
- Engraftment rate [ Time Frame: At 1 year post-transplant ]Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
- Incidence of acute graft versus host disease (GVHD) [ Time Frame: At 100 days post-transplant ]Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
- Incidence of chronic GVHD [ Time Frame: At 1 year post-transplant ]Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
- Overall survival [ Time Frame: At 1 year post-transplant ]Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson. Will be obtained using the product-limit based Kaplan-Meier method.
- Progression free survival [ Time Frame: At 1 year from time of transplant ]Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson. Will be obtained using the product-limit based Kaplan-Meier method.
- Transplant related mortality [ Time Frame: At 1 year post-transplant ]
- Immune reconstitution [ Time Frame: Up to 1 year ]Will be assessed by bone marrow transplantation SOC immunophenotyping panel and by analysis of cytomegalovirus-specific immunity.
- Myeloid and lymphoid chimerism expressed as a percentage of donor cells [ Time Frame: At 30 days ]
- Myeloid and lymphoid chimerism expressed as a percentage of donor cells [ Time Frame: At 100 days ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03333486
|Contact: ASK RPCIfirstname.lastname@example.org|
|United States, New York|
|Roswell Park Cancer Institute||Recruiting|
|Buffalo, New York, United States, 14263|
|Contact: Sophia R. Balderman 716-845-6972 Sophia.Balderman@roswellpark.org|
|Principal Investigator: Sophia R. Balderman|
|Principal Investigator:||Sophia Balderman||Roswell Park Cancer Institute|