Cord Blood Transplant for Blood Cancers

Recruiting in Montréal (>99 mi)

Overseen By Sandra Cohen, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ciusss de L'Est de l'Île de Montréal

Disqualifiers: Uncontrolled infection, HIV, Hepatitis B/C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

30 Participants Needed

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. ^{1 2 3 4 5}

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. ^{1 3 4 6 7}

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. ^{1 4 8 9 10}

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

My stem cell treatment meets the required cell count criteria.

I have been diagnosed with myelodysplastic syndrome.

I have been diagnosed with Acute Myeloid Leukemia.

Exclusion Criteria

My cancer has spread to my brain or spinal cord.

My recent bone marrow test shows a high number of immature blood cells.

I am scheduled for a transplant with a fully matched donor.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning Regimen

Patients receive an ablative conditioning regimen prior to transplantation

1-2 weeks

Transplantation

Infusion of ECT-001 expanded cord blood, which may be fresh or cryopreserved

1 day

Engraftment and Initial Recovery

Monitoring for neutrophil and platelet engraftment, and initial recovery post-transplant

6 weeks

Follow-up

Participants are monitored for safety, effectiveness, and incidence of GVHD and other complications

3 years

Treatment Details

Interventions

• Transplant with an expanded ECT-001 cord blood

Trial Overview The study tests the effectiveness of transplanting cord blood that has been expanded using ECT-001 technology in patients with aggressive blood cancers. It aims to see if this method can improve survival rates by providing better matched, higher quality stem cells than traditional methods.

Participant Groups

Group I: Main intervention Experimental Treatment 1 Intervention

Eligible patients will receive an ablative conditioning regimen and be infused with an ECT-001 expanded cord-blood. The ECT-001 expanded CB could be infused fresh, or cryopreserved.

Who Is Running the Clinical Trial?

Ciusss de L'Est de l'Île de Montréal

Lead Sponsor

Trials

81

Recruited

6,400+

Stem Cell Network

Collaborator

Trials

9

Recruited

600+

ExCellThera inc.

Industry Sponsor

Trials

5

Recruited

90+

Findings from Research

Double-unit cord blood transplantation (CBT) has significantly improved engraftment and reduced transplant-related mortality in adult patients with hematologic malignancies, addressing the limitations of single-unit CBT due to cell dose.

Despite only one unit typically sustaining donor hematopoiesis, double-unit CBT offers insights into transplant biology and may protect against relapse, making it a promising option for a wider range of patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cord blood transplants: one, two or more units? Avery, S., Barker, JN.[2010]

Cord blood transplantation (CBT) is increasingly popular in Japan, with over 1,200 procedures performed annually, and it accounts for nearly one-third of all CBTs worldwide, benefiting from factors like smaller body size and lower costs.

Recent findings indicate that while CBT has unique challenges, such as specific infectious complications and mechanisms of engraftment failure, it shows comparable survival rates to HLA-matched bone marrow or peripheral blood transplants, suggesting its efficacy as a treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cord blood transplantation in Japan. Uchida, N.[2017]

References

1. China pubmed.ncbi.nlm.nih.gov

[Successful engraftment of HLA-identical sibling cord blood transplantation in an adult with chronic myelogenous leukemia]. [2005]

2. China pubmed.ncbi.nlm.nih.gov

[Unrelated cord blood transplantation for the treatment of hematological malignancies]. [2006]

3. United States pubmed.ncbi.nlm.nih.gov

Cord blood transplants: one, two or more units? [2010]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Umbilical cord blood derived cellular therapy: advances in clinical development. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Infusion of CD3/CD28 costimulated umbilical cord blood T cells at the time of single umbilical cord blood transplantation may enhance engraftment. [2018]

6. Japan pubmed.ncbi.nlm.nih.gov

Cord blood transplantation in Japan. [2017]

7. Netherlands pubmed.ncbi.nlm.nih.gov

Ex vivo expanded cord blood cells provide rapid engraftment in fetal sheep but lack long-term engrafting potential. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Ex vivo expansion of cord blood progenitors. [2019]

9. Netherlands pubmed.ncbi.nlm.nih.gov

Ex vivo expansion, maturation, and activation of umbilical cord blood-derived T lymphocytes with IL-2, IL-12, anti-CD3, and IL-7. Potential for adoptive cellular immunotherapy post-umbilical cord blood transplantation. [2021]

10. England pubmed.ncbi.nlm.nih.gov

Advances in unrelated cord blood transplants in malignancies. [2020]