AuthorisedTherapeutic exploratory (Phase II)Interventional

A PHASE II OPEN-LABEL STUDY OF UM171-EXPANDED CORD BLOOD TRANSPLANTATION IN PATIENTS WITH HIGH AND VERY HIGH-RISK ACUTE LEUKEMIA/MYELODYSPLASIA.

This study is for adults with high-risk acute leukaemia or myelodysplasia who need a stem cell transplant. It’s looking at a new method using specially treated umbilical cord blood, called UM171-expanded cord blood. The main goal is to see how many patients remain free of their cancer one and two years after their transplant. Researchers will also check how well the new blood cells start working, whether there are serious side effects like infections or specific transplant-related problems, and how many people experience a serious reaction called graft-versus-host disease. Participation is for adults aged 18 and over, regardless of gender.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Excellthera Inc.

Enrolment target

Start

29 Nov 2024

High risk and very high risk acute leukemia/myelodysplasia requiring an allogeneic haematopoietic stem cell transplantation

What is this study about?

This study is for people who have serious blood conditions called acute leukaemia or myelodysplasia, especially if their disease is considered high-risk. These conditions mean there's a problem with the way your body makes blood cells. For some people, a treatment called a stem cell transplant (also known as a bone marrow transplant) offers the best chance of getting better.

A stem cell transplant involves replacing your unhealthy blood-forming cells with healthy ones. In this study, doctors are trying a new approach using umbilical cord blood that has been specially treated with something called UM171. This treatment aims to help the cord blood cells grow and work better after being transplanted into a patient. The main purpose of the study, which is in its second phase, is to see how effective this new method is in preventing the cancer from coming back, specifically by checking if patients are still free of their illness one and two years after the transplant.

The research team will also be carefully monitoring other important things. They’ll look at how quickly your new blood cells start working properly after the transplant, if there are any serious complications related to the transplant itself, how many people develop a reaction called graft-versus-host disease (where the new cells attack the patient’s body), and if there are any serious infections. This information will help doctors understand if this new type of cord blood transplant is a safe and effective option for patients with these challenging conditions.

Key takeaways

This study is testing a new type of stem cell transplant for serious blood cancers.
It uses specially prepared umbilical cord blood.
The main goal is to see if it helps people stay cancer-free for longer.
It's for adults (18+) with high-risk acute leukaemia or myelodysplasia.
Researchers will also track side effects and how well new blood cells grow.

Who may be eligible?

This study is open to adults who are at least 18 years old, and there is no upper age limit. Both men and women are welcome to take part.

To be considered for this study, you must have been diagnosed with acute leukaemia or myelodysplasia that is considered high-risk or very high-risk. This means your doctors believe that an allogeneic haematopoietic stem cell transplant (a type of transplant using cells from a donor) is the right treatment for you.

Your medical team will need to review your full health history to make sure that participating in this specific type of transplant study is safe and appropriate for your individual situation.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with high-risk acute leukaemia or myelodysplasia?
Do your doctors think you need a stem cell transplant from a donor?
Are you able to commit to regular follow-up appointments after the transplant?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive a stem cell transplant using the specially treated umbilical cord blood. You will have regular check-ups and tests, such as blood tests, to monitor your health and see how well the new cells are working. Doctors will also keep a close eye on any side effects you might experience and make sure you receive the best care to manage them. The study will specifically track your health for at least two years after the transplant to see if your cancer has come back and to assess any long-term effects of the treatment. The exact number of visits and tests will be explained to you by the study team, and you will have ongoing follow-up care.

Potential risks and benefits

Participating in this study might offer a chance to receive a new and potentially improved type of stem cell transplant for your condition. However, like all medical treatments, there are potential risks, including side effects from the transplant itself, infections, and a condition called graft-versus-host disease, which will be carefully monitored. The study aims to understand these risks better too. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
Netherlands

Common questions

What is 'acute leukaemia' or 'myelodysplasia'?

These are types of cancer that affect your blood and bone marrow, where new blood cells are made. They can be very serious.

What is a 'stem cell transplant'?

It's a treatment where your unhealthy blood-making cells are replaced with healthy ones, often from a donor or cord blood, to try and cure the disease.

What is 'UM171-expanded cord blood'?

It's umbilical cord blood that has been treated in a special way to help the stem cells grow and work better once they are given to you.

What does 'relapse-free survival' mean?

It means how long people in the study remain alive without their cancer coming back after their treatment.

Will I have to pay to be in this study?

No, you will not have to pay to participate in the study's treatment or tests.

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.