This clinical study is testing a new way to improve stem cell transplants for adults with high-risk blood cancers, such as leukemia or myelodysplasia, who do not have a suitable donor. The transplant uses stem cells from umbilical cord blood that have been expanded in the lab using a molecule called UM171. Previous studies showed that UM171 helps these cells grow and work better, leading to faster blood count recovery and fewer complications.
In this study, researchers are testing whether increasing the dose of UM171 during the lab expansion process can make the transplant less toxic. The hypothesis is that using a higher dose of UM171 to expand cord blood stem cells will help patients recover blood counts faster after transplant by improving the growth and function of the cells. This may lead to better immune recovery, fewer infections, shorter hospital stays, and improved overall outcomes.
Only seven patients will be enrolled, and they will be followed for one year after their transplant.
Subjects ≥ 18 and ≤ 67 years old,
Patient with either i. High risk acute leukemia or myelodysplasia defined as expected 2 year OS or PFS < 40% after a conventional allogeneic HSC transplant or ii. A hematologic malignancy requiring an allogeneic hematopoietic stem cell transplant and lack of a suitable HLA identical, haploidentical or 7/8 HLA matched donor.
A back up graft must have been identified prior to initiation of conditioning regimen.
Signed written informed consent.
Allogeneic or autologous myeloablative transplant within last 6 months.
Positive anti-donor HLA antibodies with MFI above 1500 against the selected CB (PI may approve an MFI up to 5000; if above 2500, desensitization is strongly recommended)
Use of an investigational agent within 30 days (defined as a drug not approved by Health Canada or FDA regardless of indication) of start of chemotherapy unless documented approval obtained from Sponsor.
Presence of ≥30% blasts in bone marrow or ≥ 0.5 x109/L blasts in circulating blood
Single group
ECT-001-CB
Biological/VaccineUM171-expanded cord blood
Incidence of grade 3 or higher adverse events (AEs)
Incidence of grade 3 or higher adverse events (AEs)
Feasibility of manufacturing and infusion
Proportion of selected grafts that will be expanded and meet release criteria and be successfully infused to the patient
Time to neutrophil engraftment
Neutrophil engraftment is defined as the first day of neutrophil count (ANC) ≥ 0.5 x 109/L for 3 consecutive days; first day of ANC ≥ 0.1 x 109/L will also be documented.
Incidence of NRM at 1 year post transplant
Defined as death not preceded by recurrent or progressive malignancy occurring anytime after stem cell infusion
Time to platelet engraftment
Platelet engraftment is defined as the first of 3 days of a sustained platelet count ≥ 20 x 109/L with no platelet transfusion in the preceding 7 days as per CIBMTR standards.
Incidence of graft failure
The incidence of graft failure will be monitored throughout the protocol until 1 year post transplant
Progression-free survival (PFS) at 1 year post-transplant
Relapse/progression is defined by either hematologic or extramedullary evidence of disease.
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Ciusss de L'Est de l'Île de Montréal
Lead sponsor
Stem Cell Network
Collaborator
