Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders
|Study Design:||Intervention Model: Single Group Assignment
Intervention Model Description:
This is a phase 1/phase 2 study.Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Safety and Efficacy of Miltenyi CliniMACS® CD34 Reagent System for Transplant Protocol Utilizing Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders|
- The number of patients with treatment-related serious adverse event rate (TRSAE) [ Time Frame: 42 days ]Safety will be monitored continuously with a stopping rule for toxicity based on the treatment-related serious adverse event rate (TRSAE), defined by failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft that is higher than historical control (<10% for hematologic malignancies and 34% for severe aplastic anemia).
- The number of patients with successful engraftment [ Time Frame: 42 days ]Failure defined as failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft by day 22 for patients with hematologic malignancies, and by day 42 for patients with severe aplastic anemia. Assuming a sample size of 50, if there is an engraftment success rate of 75% (or a failure rate of 25%), there would be a resulting 95% confidence interval of 63% to 87% (that is a width of 12% on either side of 75% successful engraftments
|Anticipated Study Start Date:||August 2017|
|Estimated Study Completion Date:||May 1, 2019|
|Estimated Primary Completion Date:||May 1, 2019 (Final data collection date for primary outcome measure)|
Experimental: Miltenyi CliniMACS® CD34 Reagent System
The haploidentical donor will be mobilized by G-CSF and undergo one apheresis to collect CD34+ selected stem cell product after Miltenyi CliniMACS® CD34+ selection. The products will be cryopreserved until the time of transplantation.
Recipients will receive a standard conditioning regimen. After the conditioning regimen, the subjects will receive an allograft on day 0 containing donor CD34+ cells that have been positively selected and T-cell depleted following G-CSF mobilization combined with a single UCB unit. UCB unit will not be manipulated, and will be prepared and infused separately following standard of care procedure.
Device: Miltenyi CliniMACS® CD34 Reagent System
Miltenyi CliniMACS® CD34 Reagent System will be used to prepare CD34+ enriched/T-cell depleted cells from haploidentical mobilized peripheral blood.
Please refer to this study by its ClinicalTrials.gov identifier: NCT03093844
|Contact: Constance Gorden||303-724-8649||CONSTANCE.GORDEN@UCDENVER.EDU|
|United States, Colorado|
|University of Colorado||Not yet recruiting|
|Aurora, Colorado, United States, 80045|
|Contact: Constance Gorden 303-724-8649 CONSTANCE.GORDEN@UCDENVER.EDU|
|Principal Investigator: Enkhtsetseg Purev, MD, PhD|
|Principal Investigator:||Enkhtsetseg Purev, MD, PhD||University of Colorado, Denver|