Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02566304|
Recruitment Status : Recruiting
First Posted : October 2, 2015
Last Update Posted : December 8, 2020
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukemia Acute Myeloid Leukemia in Remission Aplastic Anemia Chronic Myelomonocytic Leukemia Hodgkin Lymphoma Indolent Non-Hodgkin Lymphoma Malignant Neoplasm Myelodysplastic Syndrome Myeloproliferative Neoplasm Plasma Cell Myeloma Refractory Anemia Refractory Anemia With Excess Blasts Refractory Anemia With Ring Sideroblasts Refractory Cytopenia With Multilineage Dysplasia Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts||Drug: Fludarabine Radiation: Total-Body Irradiation Biological: T Cell-Depleted Donor Lymphocyte Infusion Drug: Cyclophosphamide Procedure: Peripheral Blood Stem Cell Transplantation Procedure: Allogeneic Hematopoietic Stem Cell Transplantation Drug: Tacrolimus Drug: Mycophenolate mofetil||Phase 2|
I. To demonstrate efficacy of this approach over the historical 2 step reduced intensity conditioning (RIC) approaches in the "vulnerable" population defined as: patients with hematopoietic cell transplant (HCT)-co-morbidity index (CI)/age scores >= 2, but no more than a score of 5 as based on the Sorror et al. data.
I. To compare the non-relapse mortality (NRM) and relapse related mortality (RRM) rates at 1 year for patients treated on this study to the that of patients undergoing haploidentical RIC hematopoietic stem cell transplantation (HSCT) as reported in the literature and as observed in the 2 step RIC trials.
II. To determine the incidence and severity of graft-versus-host disease (GVHD) in patients undergoing treated on the Thomas Jefferson University (TJU) RIC 2 step approach.
III. To evaluate engraftment rates and lymphoid reconstitution in patients treated on the TJU RIC 2 step approach.
RIC: Patients receive fludarabine phosphate intravenously (IV) over 60 minutes on days -10 to -8 and cyclophosphamide IV over 2 hours on days -3 and -2. Patients also undergo total body irradiation (TBI) followed by a donor lymphocyte infusion (DLI) on day -6.
TRANSPLANT: Patients undergo cluster of differentiation (CD)34+ peripheral blood stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus orally (PO) beginning day -1 with a taper initiated on day 42 and mycophenolate mofetil IV twice daily (BID) on days -1 to 28 in the absence of GVHD.
After completion of study treatment, patients are followed up for 1 year.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Two Step Approach to Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies|
|Actual Study Start Date :||November 13, 2015|
|Estimated Primary Completion Date :||April 23, 2021|
|Estimated Study Completion Date :||January 2022|
Experimental: RIC HSCT, GVHD prophylaxis
RIC: Patients receive fludarabine phosphate IV on days -10 to -8 and cyclophosphamide IV on days -3 and -2. Patients also undergo TBI followed by a DLI on day -7.
TRANSPLANT: Patients undergo CD34+ peripheral blood stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus PO beginning day -1 with a taper initiated on day 42 and mycophenolate mofetil IV BID on days -1 to 28 in the absence of GVHD.
Radiation: Total-Body Irradiation
Biological: T Cell-Depleted Donor Lymphocyte Infusion
Procedure: Peripheral Blood Stem Cell Transplantation
Undergo PBSC transplant
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo PBSC transplant
Drug: Mycophenolate mofetil
- Overall Survival (OS) [ Time Frame: At 1 year post HSCT ]OS will be estimated using Kaplan-Meier curves. The 1-year OS rate and corresponding 95% confidence interval will be estimated from the Kaplan-Meier curve for the OS.
- Relapse Related Mortality (RRM) [ Time Frame: At 1 year post HSCT ]Will be reported descriptively. RRM may also be estimated using Kaplan Meier curves and/or cumulative incidence analyses.
- Non-Relapse Mortality (NRM) [ Time Frame: At 1 year post HSCT ]Will be reported descriptively. NRM may also be estimated using Kaplan Meier curves and/or cumulative incidence analyses.
- Incidence and severity of GVHD [ Time Frame: Up to 1 year post HSCT ]Will be reported descriptively
- Engraftment rates [ Time Frame: Up to 1 year post HSCT ]Will be reported descriptively
- Lymphoid reconstitution [ Time Frame: Up to 1 year post HSCT ]Lymphoid reconstitution will be evaluated monthly to every other month during the first year post HSCT and will be reported descriptively.
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): NCT02566304
|Contact: Dolores Grosso, DNP, CRNP||215-955-8874|
|United States, Pennsylvania|
|Thomas Jefferson University||Recruiting|
|Philadelphia, Pennsylvania, United States, 19107|
|Contact: Dolores Grosso, DNP, CRNP 215-955-8874|
|Principal Investigator: Dolores Grosso, DNP, CRNP|
|Principal Investigator: Neal Flomenberg, MD|
|Sub-Investigator: S. Onder Alpdogan, MD|
|Sub-Investigator: Matthew Carabasi, MD|
|Sub-Investigator: Joanne Filicko-O'Hara, MD|
|Sub-Investigator: Margaret Kasner, MD|
|Sub-Investigator: Thomas Klumpp, MD|
|Sub-Investigator: William O'Hara, PharmD|
|Sub-Investigator: Ubaldo Martinez Outschoorn, MD|
|Sub-Investigator: Manish Sharma, MD|
|Sub-Investigator: John Wagner, MD|
|Sub-Investigator: Mark Weiss, MD|
|Principal Investigator:||Dolores Grosso, DNP, CRNP||Thomas Jefferson University|
|Principal Investigator:||Neal Flomenberg, MD||Thomas Jefferson University|