7 Participants Needed

UM171 for Leukemia

(ECT-001-CB-013 Trial)

SC
Overseen BySandra Cohen, Dr.
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Ciusss de L'Est de l'Île de Montréal
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new method to improve stem cell transplants for adults with high-risk blood cancers, such as leukemia, who lack a suitable donor. It involves expanding stem cells from umbilical cord blood in the lab using a molecule called UM171, a potential new drug that has shown promise in enhancing cell growth and function. Researchers aim to determine if a higher dose of UM171 can reduce transplant risks, speed up recovery, and decrease complications. Suitable candidates for this trial include individuals with high-risk leukemia or myelodysplasia who cannot find a matching stem cell donor. As a Phase 1/Phase 2 trial, this research seeks to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants the opportunity to be among the first to benefit from this innovative approach.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are using an investigational drug (a drug not approved by Health Canada or FDA), you must stop it at least 30 days before starting the trial.

Is there any evidence suggesting that UM171 is likely to be safe for humans?

Previous studies have shown that UM171-expanded cord blood transplants are safe and yield promising results for patients. Research indicates these transplants help blood cells recover faster, allowing infection-fighting and healing cells to return more quickly. This can result in fewer infections and shorter hospital stays.

Reports from past trials suggest that while some moderate cases of graft-versus-host disease (GVHD) occur, overall survival rates for patients with high-risk leukemias have been excellent. Additionally, compared to other donor transplants, UM171-expanded transplants have demonstrated better survival rates without relapse.

This information suggests that UM171 is generally well-tolerated and could improve outcomes for those needing stem cell transplants.12345

Why do researchers think this study treatment might be promising?

UM171 is unique because it offers a fresh approach to treating leukemia by utilizing an optimized dose of a new compound that enhances stem cell transplants, specifically through cord blood. Unlike traditional treatments like chemotherapy and standard stem cell transplants, UM171 modifies and amplifies the stem cells before they're transplanted, potentially increasing their effectiveness and reducing complications. Researchers are excited about UM171 because it aims to improve the success rates of transplants, offering hope for better outcomes and fewer side effects for patients with leukemia.

What evidence suggests that UM171 could be an effective treatment for leukemia?

Research has shown that UM171, the investigational treatment in this trial, can improve outcomes for patients undergoing stem cell transplants. Studies have found that UM171 enhances the growth of cord blood stem cells, leading to quicker recovery of blood cell levels. More than 80% of patients receiving these transplants had better-matched cord blood, an improvement over typical matches. The results also indicated that UM171 increased the variety of T cells, a type of immune cell, aiding in better immune system recovery. Additionally, about 70% of patients survived for two years post-transplant without disease recurrence. These findings suggest that UM171 could make stem cell transplants safer and more effective for patients with high-risk blood cancers like leukemia.12678

Who Is on the Research Team?

SC

Sandra Cohen, Dr.

Principal Investigator

CIUSSS de l'Est-de-l'Ile-de-Montréal

Are You a Good Fit for This Trial?

This trial is for adults with high-risk blood cancers like leukemia or myelodysplasia, who lack a suitable donor. Participants will undergo a stem cell transplant using cord blood treated with UM171. Specific eligibility criteria are not provided.

Inclusion Criteria

I am between 18 and 67 years old.
Signed written informed consent is required
I have a suitable cord blood unit available for treatment that meets specific health and safety standards.
See 4 more

Exclusion Criteria

I had a bone marrow transplant within the last 6 months.
I do not have severe infections, other serious cancers, HIV, hepatitis B or C, or liver cirrhosis.
I have high levels of antibodies against the donor's tissue.
See 1 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-transplant Conditioning

Participants undergo an intermediate or high-intensity conditioning regimen before receiving the transplant

1-2 weeks

Transplantation

Participants receive a single ECT-001-CB transplant with UM171-expanded cord blood stem cells

Day 0

Post-transplant Monitoring

Participants are monitored for neutrophil and platelet engraftment, immune reconstitution, and adverse events

Up to 42 days

Follow-up

Participants are monitored for safety, efficacy, and long-term outcomes including GVHD and survival

1 year

What Are the Treatments Tested in This Trial?

Interventions

  • UM171

Trial Overview

The study tests if increasing the dose of UM171 can make cord blood transplants less toxic and more effective. It aims to see if this leads to faster recovery of blood counts, better immune function, fewer infections, shorter hospital stays, and improved outcomes.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: UM171 CB transplant (ECT-001-CB) with optimized dose of UM171Experimental Treatment1 Intervention

Find a Clinic Near You

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+

Citations

Improved outcomes of UM171–expanded cord blood ...

In the phase 1-2 UM171 CB trial, more than 80% of patients received a ≥ 6/8 HLA-matched CB, which is better than the most used 5/8 matched CBs with our ...

A Study Testing an Improved Dose of UM171 to Help Make ...

Preclinical data suggest that higher UM171 concentrations may increase the number of long-term repopulating HSCs and promote balanced lymphoid ...

Long Term Follow up of UM171 Expanded Cord Blood ...

Results demonstrated that indeed UM171 overcame slow neutrophil engraftment, increased T cell receptor repertoire diversity (Dumont-Lagace M et ...

ExCellThera Announces Positive Phase 2 Study Results ...

“In these UM171 Phase 2 studies, about 70% of patients survived two years post-transplant without evidence of disease recurrence. These outcomes ...

1502 UM171 Cord Blood Transplantation for TP53 Mutated ...

The aim of this current analysis is to examine the outcome of patients with poor outcome TP53 or EVI1 anomalies who were transplanted with a single UM171 CBT.

Comparison of Outcomes after UM171-Expanded Cord ...

PFS, OS, and GRFS were all significantly better with UM171 CB than those seen with haplo donor transplants. GRFS was also consistently superior ...

UM171 Cell Therapy Earns Positive CHMP Opinion for ...

Safety profiles indicated moderate incidences of acute and chronic GVHD, with a chronic relapse-free survival rate of 55%. Approval by the ...

Paper: Infusion of UM171-Expanded Cord Blood Led to ...

Infusion of UM171-Expanded Cord Blood Led to Excellent Survival in Patients with High-Risk Leukemias: Results from Two Independent Phase II Studies