CompletedPhase IIIInterventional

MyeChild 01: Treating children with acute myeloid leukaemia

Acute myeloid leukaemia (AML) is an uncommon blood cancer in children and young people. While standard chemotherapy cures many patients, doctors want to find treatments that work better and cause fewer long-term side effects. The MyeChild 01 study is an international Phase III trial for children and teenagers under 18 who are newly diagnosed with AML. Researchers are testing different doses and combinations of chemotherapy. They are also studying a targeted medicine called gemtuzumab ozogamicin, which delivers chemotherapy directly to leukaemia cells. Some children in the trial may also be tested on different drug combinations to prepare them for a stem cell transplant. The ultimate goal is to increase cure rates, lower the chance of the cancer returning, and protect children's vital organs. Taking part is completely voluntary, and all children receive close, expert care throughout.

At a glance

Status

Completed

Phase

Phase III

Sponsor

University of Birmingham

Enrolment target

700

Start

29 Apr 2016

Estimated completion

18 Mar 2025

Acute myeloid leukaemia

What is this study about?

Acute myeloid leukaemia (AML) affects the white blood cells that normally help the body fight off infections. In AML, the bone marrow produces abnormal, immature blood cells that build up rapidly. Standard treatment involves intensive courses of chemotherapy to wipe out these leukaemia cells, but these medicines can be tough on young bodies, and the leukaemia can sometimes return.

The MyeChild 01 trial looks at ways to improve this treatment. One main focus is a medicine called gemtuzumab ozogamicin (also known as Mylotarg). This is a targeted antibody linked to a chemotherapy drug. It works like a guided missile, attaching to a specific protein on the surface of leukaemia cells and releasing its medicine directly inside them, which helps spare healthy cells.

The trial also compares different combinations of standard chemotherapy drugs to see which recipe gives the best results. For children who need a stem cell transplant to rebuild their immune system, the trial tests different conditioning treatments (chemotherapy given before the transplant) to see which is safest and most effective.

By taking part, children and their families help researchers discover clearer, gentler, and more successful ways to treat childhood AML in the NHS and across the world.

Key takeaways

MyeChild 01 is an international trial testing improved chemotherapy combinations for children and young people with AML.
It studies whether adding the targeted drug gemtuzumab ozogamicin improves cure rates and reduces relapses.
It also investigates the safest and most effective drug combinations before a stem cell transplant.
Open to patients from birth up to 17 years old who have not yet had AML chemotherapy.
Participation is completely voluntary and requires parental consent.

Who may be eligible?

This trial is open to children and young people aged from birth up to 17 years old who have just been diagnosed with acute myeloid leukaemia (AML), high-risk myelodysplastic syndrome (MDS), or a related condition called isolated myeloid sarcoma.

To be able to join, the child must not have had previous chemotherapy for AML, except for very specific emergency treatments allowed by the trial plan. Their heart, kidneys, and liver must also be working well enough to handle chemotherapy safely, which doctors check using routine tests before treatment starts.

For teenagers who could become pregnant, a negative pregnancy test is required before starting, and reliable contraception must be used during treatment. A parent or legal guardian must provide written informed consent, and young people will also be asked for their agreement (assent) if they are old enough to understand.

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.

The patient is under 18 years of age.
The patient has a confirmed new diagnosis of AML, high-risk MDS, or myeloid sarcoma.
The patient has not yet started standard chemotherapy for this leukaemia.
Heart, kidney, and liver function tests are within safe levels.
A parent or guardian agrees to give written permission for the trial.

Answer every question to see your result.

What does participation involve?

Taking part involves being admitted to a specialist children's cancer centre for several blocks of chemotherapy. Treatment is given through a central line (a soft tube placed into a vein), and each cycle requires a hospital stay of several weeks while blood counts recover. Doctors will carry out regular checks, including blood tests, heart scans (echocardiograms), and bone marrow tests (usually done under a short general anaesthetic). If a stem cell transplant is needed, this will also take place in hospital. After treatment finishes, your child will attend regular outpatient clinic visits for several years so doctors can monitor their recovery, check their organs, and ensure the leukaemia has not returned.

Potential risks and benefits

The possible benefit of joining is receiving a newer targeted medicine and helping doctors find the best treatments for children with AML in the future. However, there is no guarantee this trial treatment will work better than standard care. Chemotherapy medicines carry known side effects, such as a high risk of infections, sickness, tiredness, temporary hair loss, and mouth sores. Gemtuzumab ozogamicin can also cause changes to how the liver works. Your child's medical team will watch them very closely and give supportive medicines, like antibiotics and anti-sickness drugs, to keep them safe and comfortable. Taking part is voluntary, and you can withdraw your child from the study at any point without changing their right to standard NHS care.

Locations (1)

University of Birmingham
Approximate
Birmingham, England

Common questions

What is gemtuzumab ozogamicin?

It is a targeted therapy designed to stick to a protein found on AML cells. Once attached, it delivers a small dose of chemotherapy straight into the cancer cell.

Will my child definitely get the new drug?

Not necessarily. Because this is a randomised trial, a computer system decides which treatment group your child enters to make sure the comparison is fair.

Will my child need to stay in hospital?

Yes. Intensive chemotherapy for leukaemia requires planned hospital stays for each treatment block to keep your child safe from infections.

Does my child have to have a stem cell transplant?

No, not all children need a transplant. A transplant is only recommended if tests show the leukaemia has a higher risk of coming back.

What happens if we decide not to join the trial?

Your child will still receive standard NHS treatment for AML, which is high-quality, intensive care planned by their specialist leukaemia team.

How to find out more

Paul Wetherell

myechild01@trials.bham.ac.uk +44 121 415 1049 Official trial record

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