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Study of UM171-expanded cord blood transplantation for patients with high-risk and very high-risk acute leukemia or myelodysplasia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with high-risk acute leukemia and myelodysplasia who require a stem cell transplantation. These are serious blood disorders where abnormal cells develop in the bone marrow, affecting normal blood cell production. The treatment being studied is called ECT-001-CB, which consists of specially processed blood-forming cells derived from umbilical cord blood.

The purpose of this research is to examine how safe and effective cord blood transplantation is when the blood cells have been treated with a special expansion process. The treatment involves giving patients an intravenous infusion of these processed cord blood cells. The study will look at how well patients recover after receiving the transplant and track their health for up to two years afterward.

During the study, patients will receive the cord blood cells that have been processed using a technique to increase their number. After the transplant, doctors will monitor how well the new cells establish themselves in the patient's body and check for any complications. The study will track various aspects of recovery, including how quickly the blood counts return to normal and whether there are any immune system reactions.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment preparation
You will receive a cord blood transplant treatment. This involves using blood cells from umbilical cords that have been specially processed and expanded.
The treatment uses a specific type of blood cells called dorocubicel, which are derived from umbilical cord blood.
Step 2
Administration method
The treatment will be administered through an intravenous infusion (directly into your vein).
You will receive a single dose of expanded cord blood cells.
Step 3
Monitoring period
Your progress will be monitored for up to 2 years after the transplant.
Medical staff will check for several important factors:
- How well your body accepts the new blood cells
- Your blood cell count recovery
- Any signs of infection
- Any complications that may develop
Regular check-ups will track your recovery and response to the treatment.
Step 4
Follow-up evaluations
Medical assessments will focus on:
- Whether the disease returns (relapse-free survival)
- How well new blood cells grow (hematopoietic engraftment)
- Any complications that might occur
These evaluations will continue throughout the 2-year study period.

Who can join the trial?

9 criteria

Age between 18 and 70 years old
Diagnosed with a high or very high-risk blood cancer
Must have two matching cord blood units available that meet specific tissue typing requirements
Karnofsky score of 70 or higher (a measure of a person's ability to perform daily activities)
Heart function test showing ejection fraction of at least 40% (a measure of how well your heart pumps blood) or fractional shortening above 22%
Lung function tests showing at least 50% of normal function, including: - Forced vital capacity (amount of air you can exhale) - Forced expiratory volume (amount of air you can exhale in one second) - Diffusing capacity (ability of oxygen to pass from lungs to blood)
Liver function tests within acceptable ranges: - Bilirubin less than 2 times normal - Liver enzymes (AST and ALT) less than 2.5 times normal - Alkaline phosphatase less than 5 times normal
Kidney function showing: - Creatinine less than 2.0 mg/dl - For those with higher creatinine levels, kidney filtration rate must be above 50 ml/min
Overall health assessment score (HCT-CI) must be: - 3 or less for patients with 5% or more cancer cells in bone marrow - 5 or less for patients aged 60-70 years

Who cannot join the trial?

14 criteria

Active uncontrolled infection that could affect treatment safety
Severe heart problems, including heart failure or unstable heart disease
Severe liver problems, including liver failure (when liver cannot perform its normal functions)
Severe kidney problems, including kidney failure (when kidneys cannot filter waste from blood properly)
Pregnant or breastfeeding women
Previous stem cell transplantation
Active or uncontrolled autoimmune diseases (conditions where immune system attacks healthy cells)
HIV positive status
Active hepatitis B or C infection
Mental conditions that could interfere with understanding or following study procedures
Participation in other clinical trials within the last 30 days
Known allergies to study medications
Uncontrolled high blood pressure
Active secondary cancer (cancer that has spread from another part of the body)

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Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands

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Investigated drugs

ECT-001
is an experimental therapy used to expand cord blood cells before transplantation. It involves treating donated umbilical cord blood with a molecule called UM171 to increase the number of blood-forming stem cells. This process aims to improve the success of cord blood transplants in patients with leukemia and other blood disorders.
Cord Blood Transplantation
is a procedure where blood-forming stem cells from umbilical cord blood are given to patients to replace their diseased blood cells. The transplanted cells can help create new, healthy blood cells in the patient's body. This therapy is commonly used to treat various blood cancers and disorders.

What is already known about the treatment

ECT-001 - An experimental cell therapy treatment that involves expanding cord blood stem cells for transplantation in patients with high-risk leukemia and myelodysplasia. This therapy is administered through intravenous infusion of laboratory-expanded cord blood cells, which are processed using the ECT-001 technology to increase the number of stem cells available for transplantation. The treatment is currently being investigated in Phase II clinical trials for patients who require allogeneic hematopoietic stem cell transplantation, with the goal of improving transplant success rates and reducing the risk of graft failure. ECT-001 belongs to the class of cellular therapies and works by providing an enhanced source of blood-forming stem cells that can regenerate the patient's blood and immune system after high-dose chemotherapy.

Investigated diseases

Acute Leukemia
A rapidly progressing blood cancer where the bone marrow produces abnormal white blood cells. These abnormal cells accumulate in the bone marrow and interfere with the production of normal blood cells. The disease develops quickly, often over days or weeks, affecting the body's ability to fight infections and maintain healthy blood circulation.
Myelodysplasia
A group of blood disorders where the bone marrow produces abnormal blood cells. The condition causes the blood cells to be poorly formed or not function properly. Over time, there is a decrease in the number of healthy blood cells in the body, leading to various health issues such as fatigue and frequent infections.
Graft Versus Host Disease (GVHD)
A condition that occurs when transplanted stem cells recognize the recipient's body as foreign. The transplanted cells mount an immune response against the recipient's tissues. This can affect various organs and tissues in the body, causing different symptoms depending on which organs are involved.