Clinical trial NCT03093844: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Hematologic Disorders
Keywords: Stem Cell Transplant
Umbilical Cord Blood Transplant
Haploidentical CD34+ Selected Cells
Lead sponsor: University of Colorado, Denver
Sponsor class: OTHER
First posted: 2017-03-28
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is a phase 1/phase 2 study.
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: The number of patients with treatment-related serious adverse event rate (TRSAE)
timeFrame: 42 days
measure: The number of patients with successful engraftment
timeFrame: 42 days
Secondary outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
Study officials:
affiliation: University of Colorado, Denver
name: Jonathan Gutman
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.
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.
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.