Cord Blood Transplant for Blood Cancers
Trial Summary
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, since this is a stem cell transplant trial, it's possible that some medications might need to be adjusted or paused. Please consult with the trial team for specific guidance.
What data supports the effectiveness of the treatment Transplant with an expanded ECT-001 cord blood for blood cancers?
Research shows that cord blood transplants, which involve using blood from a newborn's umbilical cord, can be effective for treating blood cancers. Cord blood has a high number of special cells that can grow into different types of blood cells, making it a good option for transplants. Studies have shown that using cord blood can lead to successful engraftment (when the transplanted cells start to grow and make healthy blood cells) and may have fewer complications compared to other sources.12345
Is cord blood transplant generally safe for humans?
Cord blood transplants have been used safely in many patients, with some risks like infections and complications similar to other types of transplants. In Japan, studies have shown that the safety of cord blood transplants is comparable to other transplant methods, with unique infection risks that are being managed.13467
How is the treatment with expanded ECT-001 cord blood different from other treatments for blood cancers?
The treatment with expanded ECT-001 cord blood is unique because it involves expanding the number of stem cells from cord blood outside the body before transplanting them, which allows for its use in adults who need more cells than children. This approach can lead to better engraftment (successful integration of transplanted cells) and potentially improved outcomes compared to traditional cord blood transplants.148910
Research Team
Sandra Cohen, MD
Principal Investigator
Ciusss de L'Est de l'Île de Montréal
Eligibility Criteria
This trial is for adults aged 18-70 with high-risk acute leukemia or myelodysplasia, who have had inadequate responses to previous treatments or relapsed after transplants. Participants must meet specific health criteria including organ function and a Karnofsky score of at least 70%, indicating they are able to care for themselves.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Conditioning Regimen
Patients receive an ablative conditioning regimen prior to transplantation
Transplantation
Infusion of ECT-001 expanded cord blood, which may be fresh or cryopreserved
Engraftment and Initial Recovery
Monitoring for neutrophil and platelet engraftment, and initial recovery post-transplant
Follow-up
Participants are monitored for safety, effectiveness, and incidence of GVHD and other complications
Treatment Details
Interventions
- Transplant with an expanded ECT-001 cord blood
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ciusss de L'Est de l'Île de Montréal
Lead Sponsor
Stem Cell Network
Collaborator
ExCellThera inc.
Industry Sponsor