Cord Blood Transplant for Blood Cancers

Not currently recruiting at 1 trial location

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new method of using cord blood to treat certain blood cancers, such as high-risk acute leukemia and myelodysplasia. The researchers aim to evaluate the treatment's effectiveness and its impact on reducing relapse risk and chronic side effects. Participants will receive a cord blood transplant expanded using a special technique (Transplant with an expanded ECT-001 cord blood) to enhance outcomes. Individuals who have relapsed after a previous transplant or have high-risk blood cancer might be suitable candidates. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant advancements in cancer treatment.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that the ECT-001 expanded cord blood transplant is promising in terms of safety. In an earlier study, patients who received this treatment had a low risk of serious complications. Specifically, only 5% of patients experienced transplant-related death, and 10% had serious acute graft versus host disease (GVHD), where the transplanted cells attack the body. Importantly, no patients had moderate to severe chronic GVHD. Additionally, the study reported impressive survival rates, with 90% of patients surviving at least 12 months.

These findings suggest that the ECT-001 expanded cord blood transplant is generally well-tolerated by patients. This safety profile is especially encouraging for those considering joining a clinical trial with this treatment option. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for blood cancers, like leukemia, often use bone marrow transplants or chemotherapy to target cancer cells. However, this new approach with the ECT-001 expanded cord blood transplant is unique because it uses cord blood, which can be expanded to increase the number of stem cells available for transplantation. This potentially makes it easier to find matches and helps the body recover faster after treatment. Researchers are excited because it could offer a more effective and quicker recovery option than traditional methods, which often involve longer and more complex procedures.

What evidence suggests that this transplant with an expanded ECT-001 cord blood could be effective for blood cancers?

Research has shown that ECT-001 expanded cord blood transplants could be promising for treating blood cancers like acute leukemia and myelodysplasia. In a previous study, patients who received this treatment had a high survival rate, with 90% still alive after 12 months. The risk of transplant-related death was low at 5%, and severe graft versus host disease (GVHD) was uncommon. This treatment accelerated blood cell recovery and proved effective even for patients who had not succeeded with other transplants. The low progression rate of 20% in high-risk patients suggests a strong effect against leukemia. Overall, these findings support the potential effectiveness of ECT-001 expanded cord blood transplants. ^{1 2 3 4 5}

Who Is on the Research Team?

Sandra Cohen, MD

Principal Investigator

Ciusss de L'Est de l'Île de Montréal

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03441958?spons=COVERAGE%5BFullMatch%5DEXPANSION%5BNone%5D(%22ExCellThera%20inc.%22)&viewType=Table&rank=4

NCT03441958 | ECT-001 (UM171) Expanded Cord Blood ... In this new trial, the goal is to evaluate the safety and efficacy of ECT-001 expanded CB transplant in high risk MM patients. Detailed Description. This is ...

2. excellthera.com excellthera.com/news/um171-cell-therapy-demonstrates-improved-outcomes-compared-to-cord-and-matched-unrelated-donor-peripheral-blood-transplants-in-real-world-setting/

UM171 cell therapy demonstrates improved outcomes ... ECT-001 Cell Therapy demonstrated improved outcomes compared to cord blood (CB) and matched unrelated donor (MUD) peripheral blood stem cell (PBSC) transplants.

3. targetedonc.com targetedonc.com/view/umbilical-cord-blood-transplant-effective-in-patients-with-blood-cancer

Umbilical Cord Blood Transplant Effective in Patients With ... Unrelated cord blood transplant demonstrated efficacy in patients with blood cancer after microcystic adnexal carcinoma.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11287950/

Umbilical cord blood derived cell expansion - PubMed Central Currently there are 36 registered clinical trials that are examining the efficacy of expanded UCB cells with 31 of these being for haematological malignancies.

5. mycancergenome.org mycancergenome.org/content/drugs/ect-001-expanded-cord-blood/

ect-001 expanded cord blood Upon administration, the ECT-001 expanded cord blood cells increase and restore the number of hematopoietic stem and progenitor cells (HSPCs) that can ...