UM171 Expanded Cord Blood In Patients With High-Risk Acute Leukemia/Myelodysplasia

Official Title

A Phase II Open-label Study of ECT-001-expanded Cord Blood Transplantation in Patients With High-risk Acute Leukemia/Myelodysplasia

Summary:

Allogeneic hematopoietic stem cell transplantation is a life-saving procedure in patients with blood cancers. Cord blood (CB) represents an alternative source of stem cells, which is associated with a lower risk of relapse, especially in the presence of minimal residual disease in the setting of acute leukemia and myelodysplasia. Furthermore, CB has the added advantage of being associated with a low risk of chronic graft versus host disease (GVHD). Unfortunately, CB transplants are hampered by a higher risk of transplant related mortality (TRM) when compared to bone marrow/peripheral blood transplants because of the limited cell dose of CB. In the previous UM171 trial (NCT02668315), the CB expansion protocol using the ECT-001-CB technology (UM171 molecule) has proven to be technically feasible and safe. UM171 expanded CB was associated with a median neutrophil recovery at day (D)+18 post transplant. Amongst 22 patients who received a single UM171 CB transplant with a median follow-up of 18 months, risk of TRM (5%) and grade 3-4 acute GVHD (10%) were low. There was no moderate-severe chronic GVHD. Thus, overall and progression free survival at 12 months were impressive at 90% and 74%, respectively. The UM171 expansion protocol allowed access to smaller, better HLA matched CBs as >80% of patients received a 6-7/8 HLA matched CB. Interestingly there were 5 patients who had already failed an allogeneic transplant and 5 patients with refractory/relapsed acute leukemia/aggressive lymphoma. Despite this high risk population, progression was 20% at 12 months. Hence, in this new trial, investigators are targeting patients with high and very high-risk acute leukemia/myelodysplasia to test the antileukemia effect of this new graft, a UM171 expanded CB.

Trial Description

Primary Outcome:

  • Transplant Related Mortality (TRM)
  • Relapse Free survival (RFS)
  • Overall survival (OS)
Secondary Outcome:
  • Neutrophil Engraftment
  • Graft failure
  • Platelet Engraftment
  • Incidence of Acute Graft Versus Host Disease (aGVHD)
  • Incidence of Chronic Graft Versus Host Disease (cGVHD)
  • Adverse events grade 3 or higher
  • Incidence of severe infectious complications.
  • Hospitalization events
  • Incidence of preengraftment/engraftment syndrome (ES) requiring therapy.

Methodology: This is a multi-centre open label phase II clinical trial. Patients with high and very high-risk acute leukemia/myelodysplasia will receive a single 5-7/8 HLA matched ECT-001 (UM171) expanded cord blood after an ablative conditioning regimen. This group of patients would be expected to have poor progression free survival (PFS) after a conventional allogeneic transplant (bone marrow-peripheral blood).

Investigators key primary and secondary objectives include:

  • To confirm low Transplant Related Mortality (TRM)
  • To evaluate relapse free survival (RFS)
  • To analyze kinetics of hematologic engraftment;
  • To evaluate the incidence of acute and chronic GVHD
  • To evaluate the safety of the procedure
  • To evaluate incidence of infectious complications
  • To analyze duration of hospitalization
  • To evaluate the incidence of pre-engraftment/engraftment syndrome (PES/ES)
  • To analyze the effect of cryopreservation of the expanded CD34+ fraction on safety and efficacy endpoints

View this trial on ClinicalTrials.gov

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Resources

Canadian Cancer Society

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