Clinical trials

Clinical trials are research studies involving patients, that aim to find out if a new treatment is safe and effective. As well as the benefit of bringing new treatments into the clinic for mainstream use, clinical trials may offer the chance to access therapies which are outside of standard care. For certain neuroblastoma patients whose disease is advanced or does not respond well to standard treatment, enrolling onto a clinical trial can be an added option to overcome their illness.

How do clinical trials work?

Clinical trials progress through four phases to bring a new treatment to market by testing effectiveness, suitable dosage and looking for side effects.

Phase I: This is usually the first time the treatment has been trialled in people. It is usually tested on a small group of people; some may be healthy volunteers. The main aim of a Phase I trial is to test the safety of a medicine and look for any initial side effects.

Phase II: Phase II trials involve larger groups of people who have the illness that is being targeted. At this stage, the aim is to find the appropriate dose of the treatment and find out whether it is effective against the targeted disease. In the case of neuroblastoma, researchers would be looking at the tumour's response to the therapy.

Phase III: Following a successful Phase II trial, the treatment will be tested in a larger population of people with the disease of interest. It will often be tested against an existing treatment or a placebo to see if it is better in practice, and to monitor any side effects. If a treatment is successful in a Phase III trial, it will then move forward for approval into the clinic by the appropriate governing bodies.

Phase IV: After a new treatment has been approved, the safety, effectiveness and side effects will continue to be monitored whilst it is being used in practice. This is not always necessary for every new treatment.

How do I know if a clinical trial is right for my child?

You should always listen to the advice of your doctors if you are considering a clinical trial as a treatment option for your child. The options available to you will depend on several factors including the type of neuroblastoma your child has. More information about participating in clinical trials can be found on the NHS website.

Our Family Support Service is also available to offer impartial advice if you are struggling with making decisions around your child’s care.