Haploidentical Stem Cell Selection using Miltenyi CliniMACS CD34 Reagent System in Treating Participants with High-Risk Hematologic Disorders

Status: Active

Description

This phase I / II trial studies the side effects and how well haploidentical stem cells selected using Miltenyi CliniMACS CD34 reagent system works in treating participants with high-risk hematologic disorders. Stem cells selected using Miltenyi CliniMACS CD34 reagent system from a half-matched donor, may increase the number of cells given to participants during stem cell transplantation in combination with umbilical cord blood transplantation.

Eligibility Criteria

Inclusion Criteria

  • RECIPIENT INCLUSION CRITERIA
  • Diagnosed with high risk hematologic disorders warranting stem cell transplant per institutional standard of care
  • Lack human leukocyte antigen (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 total nucleated cell (TNC) (prior to thawing) of at least 2x10^7 cells per kilogram of recipient body weight
  • Ability to comprehend the nature of the treatment
  • DONOR ELIGIBILITY
  • Follow standard of care donor eligibility procedure, outlined in the standard operation procedure (SOP)
  • Deferral of donors that: * Have traveled to active Zika virus zones * Are at potential risk of transmissible spongiform encephalopathy (TSE), including Creutzfeldt–Jakob disease (CJD), based on family and travel history

Exclusion Criteria

  • RECIPIENT EXCLUSION CRITERIA
  • 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

Locations & Contacts

Colorado

Aurora
University of Colorado Hospital
Status: Active
Contact: Constance Gorden
Phone: 303-724-8649
Email: constance.gorden@ucdenver.edu

Trial Objectives and Outline

PRIMARY OBJECTIVES:

I. To determine safety and efficacy of Miltenyi CliniMACS CD34 reagent system to select haploidentical CD34+ cells.

OUTLINE: Participants are assigned to 1 of 4 groups.

GROUP I: Participants with hematologic malignancies receive fludarabine phosphate intravenously (IV) once daily (QD) on days -6 to -2, cyclophosphamide IV QD on day -6, thiotepa IV QD on days -5 to -4, and undergo total body irradiation (TBI) on days -2 to -1. Participants then undergo umbilical cord blood transplant and haploidentical hematopoietic cell transplantation on day 0 with stem cells selected using CliniMACS CD34 reagent system. Participants also receive cyclosporine IV twice daily (BID) or orally (PO) BID starting day -3 for at least 6 months and mycophenolate mofetil PO daily or BID or IV daily stating day 0 to day 30 with taper.

GROUP II: Participants with hematologic malignancies who cannot tolerate high intensity regimen receive fludarabine phosphate IV QD on days -6 to -2, cyclophosphamide IV QD on day -6, and undergo TBI on -1. Participants then undergo umbilical cord blood transplant and haploidentical hematopoietic cell transplantation on day 0 with stem cells selected using CliniMACS CD34 reagent system. Participants also receive cyclosporine IV BID or PO BID starting day -3 for at least 6 months and mycophenolate mofetil PO daily or BID or IV daily stating day 0 to day 30 with taper.

GROUP III (HIGH DOSE TBI): Participants with hematologic malignancies receive fludarabine phosphate IV QD on days -7 to -6, cyclophosphamide IV QD on days -6 and -5, and undergo TBI BID days -3 to -1. Participants then undergo umbilical cord blood transplant and haploidentical hematopoietic cell transplantation on day 0 with stem cells selected using CliniMACS CD34 reagent system. Participants also receive cyclosporine IV BID or PO BID starting day -3 for at least 6 months and mycophenolate mofetil PO daily or BID or IV daily stating day 0 to day 30 with taper.

GROUP IV: Participants with aplastic anemia receive cyclophosphamide IV QD on days -7 and -6, fludarabine phosphate IV QD on days -5 to -1, anti-thymocyte globulin on days -5 to -2, and undergo TBI on day -1. Participants then undergo umbilical cord blood transplant and haploidentical hematopoietic cell transplantation on day 0 with stem cells selected using CliniMACS CD34 reagent system. Participants also receive cyclosporine IV BID or PO BID starting day -3 for at least 6 months and mycophenolate mofetil PO daily or BID or IV daily stating day 0 to day 30 with taper.

After completion of study treatment, participants are followed up at 60 and 100 days.

Trial Phase & Type

Trial Phase

Phase I/II

Trial Type

Treatment

Lead Organization

Lead Organization
University of Colorado Hospital

Principal Investigator
Enkhtsetseg Purev

Trial IDs

Primary ID 16-1672
Secondary IDs NCI-2018-01145
Clinicaltrials.gov ID NCT03093844