CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
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|ClinicalTrials.gov Identifier: NCT01049854|
Recruitment Status : Completed
First Posted : January 15, 2010
Last Update Posted : August 21, 2018
|Condition or disease||Intervention/treatment||Phase|
|Leukemia Lymphoma Bone Marrow Failure Immunodeficiencies Histiocytosis Sickle Cell Disease Beta Thalassemia Inborn Errors of Metabolism||Drug: Full Intensity with TBI Drug: Full Intensity Drug: Reduced Intensity Drug: Reduced Intensity (Fanconi)||Phase 2|
The selection of CD34+ cells is associated with the simultaneous depletion of T cells that are responsible for severe acute and chronic graft versus host disease (GVHD). Successful engraftment is reported in adult patients with malignant and non-malignant disease who received CD34+ selected stem cells from HLA-matched or mismatched mobilized peripheral blood (PBSC) or bone marrow.
Selected patients defined in the eligibility criteria will enrolled on this study. Patients will receive one of either full intensity or reduced intensity regimen based on the patient's disease status, organ function and performance and determined by the PI and will have peripheral blood undergo CD34 selection.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||CD34+Stem Cell Selection for Patients Receiving Partially Matched Family or Matched Unrelated Adult Donor Allogeneic Stem Cell Transplantations for Malignant and Non-Malignant Disease|
|Study Start Date :||September 2011|
|Actual Primary Completion Date :||November 2017|
|Actual Study Completion Date :||August 2018|
Full intensity with TBI
Drug: Full Intensity with TBI
Patients will start their pre-conditioning regimen on Day -8. Fractionated TBI will be administered twice daily for 3 days on Days -8, -7, and -6. Patients will receive Thiotepa on Days -5, -4, Cyclophosphamide on Days -3, -2 and rabbit antithymocyte globulin on Days -4, -3, -2 and -1. The stem cell infusion will be performed on Day 0. GM-CSF hematopoietic growth factor will start on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Full intensity without TBI
Drug: Full Intensity
Patients will start their pre-conditioning regimen on Day -9. Patients will receive busulfan twice daily on Days - 8, -7, -6, and -5 and Melphalan on Days -4, -3 and -2 and rabbit antithymocyte globulin on Days -4, -3, -2 and -1 with stem cell infusion on Day 0. GM-CSF hematopoietic growth factor will start on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Reduced Intensity Chemotherapy
Drug: Reduced Intensity
Patients will start their GVHD prophylaxis with Tacrolimus on Day -9. Patients will receive busulfan twice daily on Days -8, -7, -6, and -5; fludarabine on Days -7, -6, -5, -4, -3 and -2 and alemtuzumab on Days -5, -4, -3, -2, and -1. The stem cell infusion will be performed on Day 0. GVHD prophylaxis will consist of tacrolimus only.
Reduced Intensity Chemotherapy for Fanconi Anemia
Drug: Reduced Intensity (Fanconi)
Patients will start their pre-conditioning regimen on Day -6. Patients will receive TBI as a single fraction on Day -6. Patients will receive fludarabine and cyclophosphamide on Days - 5, -4, -3, and -2 and antithymocyte globulin (horse) on Days -5, -4, -3, -2 and -1. The stem cell infusion will be performed on Day 0. GVHD prophylaxis will consist of tacrolimus only.
- The safety CD34+ stem cell selection [ Time Frame: 100 days ]serious adverse events will be monitored post transplant to determine if there is an increase vs. historical data related to the CD34+ selection
- Immune reconstitution (T, B, DC) following CD34+ selection [ Time Frame: 3 years ]immune subsets will be drawn post transplant to determine the rate of reconstitution post CD34+ transplant to determine if this process increases or decreases the reconstitution time.
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): NCT01049854
|United States, New York|
|New York Medical College|
|Valhalla, New York, United States, 10595|
|Principal Investigator:||Mitchell S Cairo, MD||New York Medical College|