Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03093844|
Recruitment Status : Active, not recruiting
First Posted : March 28, 2017
Last Update Posted : December 10, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hematologic Disorders||Device: Miltenyi CliniMACS® CD34 Reagent System||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||200 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||This is a phase 1/phase 2 study.|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||October 12, 2017|
|Estimated Primary Completion Date :||February 10, 2022|
|Estimated Study Completion Date :||February 10, 2022|
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.
- 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
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): NCT03093844
|United States, Colorado|
|University of Colorado|
|Aurora, Colorado, United States, 80045|
|Principal Investigator:||Jonathan Gutman||University of Colorado, Denver|