Active not recruitingPHASE2, PHASE3INTERVENTIONAL

A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT

This study focuses on improving stem cell transplants for adults with acute myeloid leukaemia (AML) and high-risk myelodysplastic syndrome (MDS). While these transplants can be very effective, they can also have serious side effects, especially for older patients. Recent advances mean more older adults can now have these transplants, but the risk of the disease coming back (relapse) is still a concern, particularly for those with a more aggressive disease. Researchers are comparing different treatment approaches before and during the transplant to see if they can make the procedure safer and more successful. This includes testing new pre-transplant treatments and different combinations of medications used to prepare the body for the transplant. The goal is to help more patients achieve lasting recovery.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

University of Birmingham

Enrolment target

333

Start

27 Jan 2020

Estimated completion

01 May 2026

Acute Myeloid Leukaemia High-risk Myelodysplastic Syndrome

What is this study about?

This study is looking into new ways to make stem cell transplants more successful for adults living with certain types of blood cancer: acute myeloid leukaemia (AML) and high-risk myelodysplastic syndrome (MDS). For many years, stem cell transplants have been one of the most powerful treatments for these conditions. However, they are also quite intense treatments and can have significant side effects. Because of these side effects, older adults were often not able to have them.

In recent times, medical advances have meant that 'reduced intensity' stem cell transplants can be offered. These are gentler on the body, which has opened the door for more older patients to benefit from this potentially life-saving treatment. While this is a huge step forward, there's still a challenge: for some patients, especially those with more aggressive forms of the disease, there's a risk that the cancer might come back after the transplant.

That's where this study comes in. Doctors are trying out new treatment combinations to improve the chances of success. They are comparing different medications given before the transplant and different mixes of medicines used to prepare the body for the new stem cells. By doing this, they hope to find better ways to help patients avoid the disease returning and make the transplant experience as effective and safe as possible. All patients joining the study will be carefully monitored for at least two years to see how these new approaches work.

Key takeaways

This study explores new ways to improve stem cell transplants for AML and high-risk MDS.
It aims to make transplants safer and more effective, especially for older adults.
Researchers are testing different medication combinations before and during the transplant.
Participation involves receiving treatment according to a study plan and regular follow-ups for at least two years.
The goal is to reduce the chance of the disease returning after a successful transplant.
You can withdraw from the study at any time without affecting your ongoing care.

Who may be eligible?

This study is for adults aged 18 or over who have been diagnosed with acute myeloid leukaemia (AML) or high-risk myelodysplastic syndrome (MDS). This means you might be considered if your AML is currently in remission (meaning the signs of cancer are reduced or gone), or if you have a certain type of high-risk MDS.

To join, you would need to be considered fit enough by your doctor for a stem cell transplant and have a suitable donor for the transplant. This could be a matched family member or an unrelated donor. There are also specific health checks your medical team will do to make sure the treatment is suitable for you. You would also need to be willing to use effective contraception during the study if you are able to have children.

Some reasons why you might not be able to join include having certain kidney or liver problems, active infections like HIV or hepatitis, or if you are pregnant or breastfeeding. Your doctor will carefully review all your health information to see if this study is the right option for you.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of AML (in remission) or high-risk MDS?
Has your doctor said you are well enough for a stem cell transplant?
Do you have a suitable stem cell donor?
Are you able to use effective contraception if you or your partner could become pregnant?
Do you not have certain severe kidney, liver, or active infection conditions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be assigned to one of the treatment groups by chance. This is a bit like tossing a coin, but it’s done by a computer to ensure fairness. Which exact treatment you receive will depend on your age and your specific condition, and it might involve different combinations of medications like Vyxeos, Fludarabine, Busulphan, Thiotepa, or Cytarabine.

You will receive your treatment, including the stem cell transplant, as you normally would, but following the specific plan of the study. You will have regular check-ups, blood tests, and other assessments as needed to monitor your health and how well the treatment is working. After your transplant, your health will continue to be monitored closely for at least two years. This helps the study team understand the long-term effects of the treatments. You'll have scheduled hospital visits for these follow-ups.

Potential risks and benefits

Taking part in this study might offer you access to new and potentially more effective treatments that are not yet widely available. The goal is to improve the success of stem cell transplants and reduce the chance of your disease returning. However, like all medical treatments, there are potential risks and side effects from the medications used and the transplant itself. These will be fully explained to you by your medical team so you can make an informed decision. Remember, your participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (14)

Queen Elizabeth Hospital
Birmingham, United Kingdom
University Hospitals Bristol
Bristol, United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom
University Hospital of Wales
Cardiff, United Kingdom
Queen Elizabeth Hospital Glasgow
Glasgow, United Kingdom
St James' University Hospital
Leeds, United Kingdom
Leicester Royal Infirmary
Leicester, United Kingdom
Hammersmith Hospital
London, United Kingdom
King's College Hospital
London, United Kingdom
Manchester Royal Infirmary
Manchester, United Kingdom
Freeman Hospital
Newcastle, United Kingdom
Nottingham City Hospital
Nottingham, United Kingdom

+2 more sites — see the official record for the full list.

Common questions

What is a stem cell transplant?

A stem cell transplant replaces unhealthy blood-forming cells with healthy ones, usually from a donor, to help your body produce new, healthy blood cells.

What is AML and MDS?

AML (Acute Myeloid Leukaemia) and MDS (Myelodysplastic Syndrome) are types of blood cancers where your body doesn't make enough healthy blood cells.

Why is this study needed?

This study aims to find better ways to make stem cell transplants more successful and reduce the risk of the disease coming back, especially for older patients.

Will I get 'new' treatment if I join?

You might receive new treatment combinations being tested, or you might receive the standard treatment. Your doctor will discuss which options are relevant for you.

How long will I be followed in the study?

You will be followed up and regularly checked by the study team for at least two years after your transplant.