Clinical trials

Clinical trials are research studies that involve patients. They help doctors find out whether a new treatment is safe, what dose to use, and whether it works. Clinical trials can also give access to treatments that are not yet part of standard care. 

For some children with neuroblastoma, especially when the cancer is advanced or not responding to usual treatments, a clinical trial may provide another option. 

How do clinical trials work?

Phase I
Phase I trials are the first time a new treatment is given to people. 
They involve a small number of participants. 
The main aim is to check the safety of the treatment and watch for any early side effects. 

Phase II
Phase II trials involve a larger group of people who have the illness the treatment is designed for. 
The aim is to find the best dose and to see whether the treatment works. 
For neuroblastoma, doctors look at how the tumour responds. 

Phase III
Phase III trials include an even larger group of people with the same condition. 
The new treatment is compared with the current standard treatment or sometimes with a placebo. 
These trials help doctors understand if the new treatment works better and what side effects it may cause. 
If a Phase III trial is successful, the treatment may then be considered for approval for wider use. 

Phase IV
Phase IV trials take place after a treatment has been approved. 
Doctors continue to check long term safety, how well it works over time, and whether any new side effects appear. 
Not every treatment needs a Phase IV trial. 

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.