Childhood acute myeloid leukaemia (AML) clinical trials

Because cancer is so rare in children, clinical trials are really important for testing new treatments and improving current ones, so more children are cured and long-term side effects are reduced.

If there’s a clinical trial available that’s suitable for your child, your doctor may ask you, (or your child, if they’re over 15), to consider taking part. In a trial, your child’s safety and wellbeing are always the priority. They’ll be closely checked during and after the trial – and can withdraw at any time.

The latest national clinical trial for childhood AML is called MyeChild01. It is still running but is not accepting any new participants. The trial has several aims, including:

• to explore the effect of adding gemtuzumab ozogamicin (Mylotarg®) to induction therapy, and decide the best dosage
• to compare two types of conditioning (preparation) for a stem cell transplant
• to understand more about how measurable (or minimal) residual disease (MRD) can guide treatment decisions. MRD means the number of leukaemia cells left in the body after a course of treatment – it can be tiny but still affect remission.

Taking part in a trial does come with uncertainties and risks, and there’s no guarantee the treatment being tested will be better than the standard recommended treatment. If you don’t want your child to be in a trial, or there isn’t a suitable trial running, they will be offered the best treatment available that’s right for them, based on their test results and general health.

Your child’s hospital team can give you more information about clinical trials for childhood AML. You can also contact our Clinical Trials Support Service to find out more about trials and talk things through.