Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders

NCT03093844 | Completed Results DMC FDA Device

• 1 other identifier: 16-1672.cc
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

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.

Conditions

Hematologic Disorders

Keywords

Stem Cell Transplant; Umbilical Cord Blood Transplant; Haploidentical CD34+ Selected Cells

Outcome Measures

Primary Outcomes (1)

• The Number of Patients With Successful Engraftment

  To determine the rate of successful engraftment of patients who received haploidentical CD34+ selected cells combined with single unit umbilical cord blood transplant. Failure defined as failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft by day 42.

  42 days

Study Arms (1)

Miltenyi CliniMACS® CD34 Reagent System

EXPERIMENTAL

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

Interventions

Miltenyi CliniMACS® CD34 Reagent System DEVICE

Miltenyi CliniMACS® CD34 Reagent System will be used to prepare CD34+ enriched/T-cell depleted cells from haploidentical mobilized peripheral blood.

Miltenyi CliniMACS® CD34 Reagent System

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Colorado, Denver: lead

Study Sites (1)

University of Colorado

Aurora, Colorado, 80045, United States

Location

Related Publications (4)

• 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.

  PMID: 27788764 BACKGROUND

• 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.

  PMID: 27333804 BACKGROUND

• 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.

  PMID: 16670070 BACKGROUND

• 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.

  PMID: 25255162 BACKGROUND

MeSH Terms

Conditions

Hematologic Diseases

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Dr. Jonathan Gutman
• Organization: University of Colorado

JONATHAN.GUTMAN@CUANSCHUTZ.EDU

303-724-9562

Study Officials

• Jonathan Gutman

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a phase 1/phase 2 study.

Study Record Dates

• First Submitted: March 22, 2017
• First Posted: March 28, 2017
• Study Start: October 12, 2017
• Primary Completion: November 27, 2024
• Study Completion: February 5, 2025
• Last Updated: August 15, 2025
• Results First Posted: August 15, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)