Back to resultsHaploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders
Primary Purpose
Hematologic Disorders
Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Miltenyi CliniMACS® CD34 Reagent System
Sponsored by
About this trial
This is an interventional treatment trial for Hematologic Disorders focused on measuring Stem Cell Transplant, Umbilical Cord Blood Transplant, Haploidentical CD34+ Selected Cells
Eligibility Criteria
Inclusion Criteria - Recipient
- Ages 18-80 years inclusive
- Diagnosed with high risk hematologic disorders warranting stem cell transplant per institutional standard of care
- Lack HLA-identical related donor
- Availability of at least one HLA- haploidentical (i.e. => 5/10 and <= 8/10 HLA match) related donor (HLA-A, B, C, DR, and DQ loci) who is available to donate CD34+ cells.
- Availability of at least one 4/6 HLA-matched (HLA-A, B, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP). The cord blood unit must contain a minimum TNC (prior to thawing) of at least 2x107 cells per kilogram of recipient body weight
- Ability to comprehend the nature of the treatment 6.2. Exclusion Criteria - Recipient (any of the following)
- HLA identical (6/6) related donor available and readily accessible at time of transplantation evaluation
- Any patient not meeting institutional standard guidelines for transplant eligibility
Sites / Locations
- University of ColoradoRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Miltenyi CliniMACS® CD34 Reagent System
Arm Description
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.
Outcomes
Primary Outcome Measures
The number of patients with treatment-related serious adverse event rate (TRSAE)
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
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
Secondary Outcome Measures
Full Information
NCT ID
NCT03093844
First Posted
March 22, 2017
Last Updated
February 24, 2023
Sponsor
University of Colorado, Denver
1. Study Identification
Unique Protocol Identification Number
NCT03093844
Brief Title
Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 12, 2017 (Actual)
Primary Completion Date
February 10, 2024 (Anticipated)
Study Completion Date
February 10, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Colorado, Denver
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
Yes
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a study to evaluate the safety and efficacy of Miltenyi CliniMACS® CD34 Reagent System to promote engraftment of haploidentical CD34+ selected cells combined with single unit umbilical cord blood transplant for treatment of high-risk hematologic disorders.
Detailed Description
In this clinical protocol, the CliniMACS® CD34 Reagent System will be used for processing hematopoietic progenitor cells collected by apheresis (HPC, Apheresis) from an allogeneic, HLA-haploidentical, related donor to obtain a CD34+ cell-enriched population for hematopoietic reconstitution. The haploidentical donor will be mobilized by G-CSF and undergo one apheresis to collect CD34+ stem cells. The products will be cryopreserved until the time of transplantation. Recipients with hematologic disorders who require transplant will receive a standard conditioning regimen and 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hematologic Disorders
Keywords
Stem Cell Transplant, Umbilical Cord Blood Transplant, Haploidentical CD34+ Selected Cells
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
This is a phase 1/phase 2 study.
Masking
None (Open Label)
Allocation
N/A
Enrollment
200 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Miltenyi CliniMACS® CD34 Reagent System
Arm Type
Experimental
Arm Description
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.
Intervention Type
Device
Intervention Name(s)
Miltenyi CliniMACS® CD34 Reagent System
Intervention Description
Miltenyi CliniMACS® CD34 Reagent System will be used to prepare CD34+ enriched/T-cell depleted cells from haploidentical mobilized peripheral blood.
Primary Outcome Measure Information:
Title
The number of patients with treatment-related serious adverse event rate (TRSAE)
Description
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).
Time Frame
42 days
Title
The number of patients with successful engraftment
Description
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
Time Frame
42 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria - Recipient
Ages 18-80 years inclusive
Diagnosed with high risk hematologic disorders warranting stem cell transplant per institutional standard of care
Lack HLA-identical related donor
Availability of at least one HLA- haploidentical (i.e. => 5/10 and <= 8/10 HLA match) related donor (HLA-A, B, C, DR, and DQ loci) who is available to donate CD34+ cells.
Availability of at least one 4/6 HLA-matched (HLA-A, B, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP). The cord blood unit must contain a minimum TNC (prior to thawing) of at least 2x107 cells per kilogram of recipient body weight
Ability to comprehend the nature of the treatment 6.2. Exclusion Criteria - Recipient (any of the following)
HLA identical (6/6) related donor available and readily accessible at time of transplantation evaluation
Any patient not meeting institutional standard guidelines for transplant eligibility
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Derek Schatz
Phone
720-848-0628
Email
derek.schatz@cuanschutz.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jonathan Gutman
Organizational Affiliation
University of Colorado, Denver
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Derek Schatz
Phone
720-848-0628
Email
derek.schatz@cuanschutz.edu
First Name & Middle Initial & Last Name & Degree
Jonathan Gutman
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
27788764
Citation
van Besien K, Childs R. Haploidentical cord transplantation-The best of both worlds. Semin Hematol. 2016 Oct;53(4):257-266. doi: 10.1053/j.seminhematol.2016.07.004. Epub 2016 Jul 25.
Results Reference
background
PubMed Identifier
27333804
Citation
van Besien K, Koshy N, Gergis U, Mayer S, Cushing M, Rennert H, Reich-Slotky R, Mark T, Pearse R, Rossi A, Phillips A, Vasovic L, Ferrante R, Hsu YM, Shore T. Cord blood chimerism and relapse after haplo-cord transplantation. Leuk Lymphoma. 2017 Feb;58(2):288-297. doi: 10.1080/10428194.2016.1190970. Epub 2016 Jun 23.
Results Reference
background
PubMed Identifier
16670070
Citation
Magro E, Regidor C, Cabrera R, Sanjuan I, Fores R, Garcia-Marco JA, Ruiz E, Gil S, Bautista G, Millan I, Madrigal A, Fernandez MN. Early hematopoietic recovery after single unit unrelated cord blood transplantation in adults supported by co-infusion of mobilized stem cells from a third party donor. Haematologica. 2006 May;91(5):640-8.
Results Reference
background
PubMed Identifier
25255162
Citation
Kwon M, Bautista G, Balsalobre P, Sanchez-Ortega I, Serrano D, Anguita J, Buno I, Fores R, Regidor C, Garcia Marco JA, Vilches C, de Pablo R, Fernandez MN, Gayoso J, Duarte R, Diez-Martin JL, Cabrera R. Haplo-cord transplantation using CD34+ cells from a third-party donor to speed engraftment in high-risk patients with hematologic disorders. Biol Blood Marrow Transplant. 2014 Dec;20(12):2015-22. doi: 10.1016/j.bbmt.2014.08.024. Epub 2014 Sep 22.
Results Reference
background
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Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders
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