Expanded Cord Blood in Patients in Need of an Allogeneic Stem Cell Transplant

Official Title

A Phase I-II Open-label Study of UM171 Expanded Cord Blood in a Fed-batch Culture System (UFCB-001) in Patients Who Need an Allogeneic Hematopoietic Stem Cell Transplant But Lack a Suitable Donor

Summary:

Allogeneic hematopoietic stem cell transplantation is a life-saving procedure in patients with blood cancers, but only 25% of transplant candidates have a sibling donor. A matched unrelated donor can be found for 60% of patients but this number is lower for non-Caucasians. Cord blood (CB), another source of stem cells, has major advantages over unrelated donors including immediate availability, better permissiveness in immune mismatches between donor and transplant recipient, better availability for non-Caucasians, and less graft versus host disease, a complication frequently seen after transplant which negatively affects quality of life. Unfortunately, the use of CB is still limited in adults because of the small number of stem cells. UM171, a molecule with hematopoietic stem cell expansion properties, has been shown to increase cord blood stem cells 13 fold. In this trial, Investigators will use UM171 treated CB in patients who need a transplant but lack an acceptable donor.This protocol seeks to test the safety of CB cells expanded with UM171, and to determine the kinetics of engraftment as well as the minimal cord blood unit cell dose that when expanded achieves prompt engraftment.

Trial Description

Primary Outcome:

  • Monitoring adverse events, toxicities and medical evolution
Secondary Outcome:
  • Feasibility of cord blood expansion using UM171 (transplant success)
  • Kinetics of hematopoietic recovery
  • Minimal cord blood cell dose that when expanded achieves prompt engraftment
  • Correlation between neutrophil and platelet engraftment and CD34+ and CD34+CD45RA- dose
  • Incidence of primary and late graft failure
  • Incidence of backup cord infusion and graft dominance
  • Incidence of acute and chronic GVHD
  • Incidence of severe infectious complications
  • T cell evaluation (Cluster of differentiation (CD) 4+ and CD8+)
  • B cell evaluation
  • Natural Killer (NK) cell evaluation
  • Transplant related mortality (TRM)
  • progression free survival
  • Overall survival
  • Incidence of engraftment syndrome requiring therapy
Investigators are proposing a phase I-II, Canadian multi-centre, open-label study of UM171 ex vivo expanded CB transplant in 25 patients who need an allogeneic hematopoietic stem cell transplantation (HSCT) but lack a Human Leucocyte Antigen (HLA) matched donor. Investigators key primary and secondary objectives include: 1. To establish the feasibility of expanding cord blood units for allogeneic cord blood transplantation. 2. To establish the safety and identify unexpected toxicities associated with the transplantation of cord blood cells expanded with UM171/fed-batch culture system. 3. To measure kinetics of neutrophil and platelet recovery. 4. To determine minimal cord blood unit cell dose (Total Nucleated Cell (TNC)/CD34+ cells) that when expanded achieves prompt engraftment as a single cord transplant. Methodology: Patients with a hematologic malignancy and an indication for allogeneic HSCT who lack a matched unrelated donor will receive a myeloablative or submyeloablative conditioning regimen followed by infusion of UM171 expanded CB graft. Accrual is expected to last 18 months and patients will be followed for 3 years. Expected benefits: Investigators expect that expansion with UM171/fed-batch will be safe and lead to both rapid and sustained engraftment. This will likely decrease the high early morbidity/mortality of CB HSCT and improve access to transplant, especially ethnic minorities. In addition, if low cell dose is solved, patients will benefit from CB's lower risk of chronic (graft versus host disease) GVHD, a major cause of morbidity.

View this trial on ClinicalTrials.gov

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Resources

Canadian Cancer Society

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