RecruitingPHASE3INTERVENTIONAL

International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia

This study, called MyeChild 01, is looking for better ways to treat children and teenagers with Acute Myeloid Leukaemia (AML), a serious blood cancer. Researchers are comparing different chemotherapy treatments to see which ones work best and have the fewest side effects. They are also trying to find the best number of doses for a drug called gemtuzumab ozogamicin when given with other chemotherapy. The study also investigates two different types of stem cell transplants, which are used for patients at higher risk of their cancer coming back. The aim is to find out if a gentler type of transplant can lead to better outcomes by reducing serious side effects, without increasing the risk of the cancer returning. The ultimate goal is to improve survival rates and the quality of life for children with AML.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

University of Birmingham

Enrolment target

700

Start

01 Apr 2016

Estimated completion

01 Dec 2032

Acute Myeloid Leukaemia

What is this study about?

When children are diagnosed with Acute Myeloid Leukaemia (AML), it's a very challenging time for families. This study, known as MyeChild 01, is an important international effort to find improved treatments for this type of blood cancer. The main idea is to compare different combinations of medicines and treatment approaches to see which ones are the most effective at fighting the cancer, while also being as safe as possible for children and teenagers.

One key part of the study is looking at different chemotherapy drugs. Researchers want to know which combinations work best as the initial treatment (called 'induction therapy'). They are also trying to figure out the right amount and number of doses for a specific drug called gemtuzumab ozogamicin, to make sure it's given safely alongside other chemotherapy. For patients whose cancer responds well to initial treatment, the study compares two different follow-up treatments (called 'consolidation regimens') to help stop the leukaemia from coming back.

For some children who are at a higher risk of their AML returning, a stem cell transplant might be an option. This study is comparing two different ways to prepare a child's body for a stem cell transplant: a standard, more intense method, and a gentler one. The hope is that the gentler method could reduce serious side effects and improve the child's recovery, without making it more likely for the cancer to return. By carefully comparing these different treatments, the study aims to improve the chances of long-term health and survival for children with AML.

Key takeaways

This study aims to improve treatments for children with AML.
It compares different chemotherapy drugs and stem cell transplant methods.
The goal is to find more effective and safer treatment options.
Participation involves receiving study treatments and regular monitoring.
You can withdraw from the study at any time.
Your child's safety and well-being are the top priority.

Who may be eligible?

To be able to join this study, a child or teenager must have been recently diagnosed with Acute Myeloid Leukaemia (AML), or a related condition called high-risk Myelodysplastic Syndrome, or a myeloid sarcoma. They need to be under 18 years old and not have had chemotherapy or other similar treatments for their condition before, unless the study rules allow for it.

It's important that their heart and other main organs are working well enough to safely receive the study treatments. For girls and young women, a pregnancy test will be needed, and if they could become pregnant, they'd need to use effective birth control. Lastly, a parent, guardian, or the patient themselves (ifold enough) would need to give written permission to join the study.

Some parts of the study, especially those involving the drug gemtuzumab ozogamicin, have slightly different age and kidney/liver function requirements. Generally, older children have more options for participating in these specific parts.

Quick self-check

Is my child under 18 years old?
Does my child have a new diagnosis of AML or a related condition?
Has my child received chemotherapy for AML before?
Is my child's heart, kidney, and liver function generally good?
Am I, or is my child (if old enough), willing to provide written consent?

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

What does participation involve?

If your child joins this study, they will receive their leukaemia treatment as part of the study plan. This means receiving chemotherapy drugs, and potentially a stem cell transplant, according to specific schedules. There will be regular visits to the hospital for treatment, check-ups, and tests like blood tests, heart scans, and kidney/liver function tests to monitor their health and how well the treatment is working.

The study will involve doctors deciding which treatment your child receives, sometimes by a process called 'randomisation' – like flipping a coin – to ensure a fair comparison between treatments. The total length of their participation will vary depending on the treatment plan, but it will involve the entire course of their initial cancer treatment, as well as follow-up appointments to track their progress and long-term health. The medical team will explain the full schedule and what to expect at each stage.

Potential risks and benefits

Taking part in this study could offer your child access to new and potentially more effective treatments for AML, which might improve their chances of getting better. Researchers hope to find treatments that are both powerful against the cancer and kinder on the body. However, like all cancer treatments, there are potential risks and side effects from the chemotherapy and stem cell transplants, which will be carefully explained by the medical team. These could include infections, fatigue, nausea, and other serious complications. Your child's care and safety will be the top priority, and you have the right to withdraw from the study at any time, for any reason, without it affecting your child's medical care.

Locations (68)

Women and Children's Hospital Adelaide
Adelaide, Australia· Recruiting
Queensland Children's Hospital
Brisbane, Australia· Recruiting
Monash Children's Hospital
Melbourne, Australia· Recruiting
Royal Childrens Hospital
Melbourne, Australia· Recruiting
John Hunter Children's Hopsital
New Lambton Heights, Australia· Recruiting
Perth Children's Hospital
Perth, Australia· Recruiting
Sydney Children's Hospital
Sydney, Australia· Recruiting
The Childrens Hospital At Westmead
Westmead, Australia· Recruiting
Centre Hospitalier Universitaire Amiens - Picardie
Amiens, France· Recruiting
Centre Hospitalier Universitaire D'angers
Angers, France· Recruiting
Centre Hospitalier Regional Universitaire Besancon - Hopital Jean Minjoz
Besançon, France· Recruiting
Centre Hospitalier Universitaire De Bordeaux - Hopital Pellegrin
Bordeaux, France· Recruiting

+56 more sites — see the official record for the full list.

Common questions

What is Acute Myeloid Leukaemia (AML)?

AML is a type of cancer that starts in the bone marrow, the soft tissue inside bones where new blood cells are made. It causes the bone marrow to produce abnormal white blood cells.

What does 'Phase III' mean for a clinical trial?

A Phase III trial is usually the final stage of testing a new treatment. It compares a new treatment with the best existing one to see which is better and safer for patients.

What is gemtuzumab ozogamicin?

It's a special type of drug that combines an antibody (to target cancer cells) with a chemotherapy drug. It's designed to deliver the chemotherapy more directly to the cancer cells.

What is a stem cell transplant?

A stem cell transplant replaces unhealthy blood-forming cells with healthy ones. It's often used for high-risk leukaemia to help the body make healthy blood cells again.

Will my child definitely get the new treatment?

Not necessarily. Sometimes treatments are chosen by chance (randomisation) to see which works best. Your doctor will explain what your child's treatment plan would involve.

How to find out more

Christina Ryan

myechild01@trials.bham.ac.uk 01214151049 Official trial record

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