A clinical trial is a scientific study, or an organised test of medicines and new treatment options involving patient volunteers. Clinical trials confirm whether medicines are safe and effective to introduce as new treatments for a particular disease or condition. All clinical trials need to be approved by the ethics committee of each participating institution before a trial commences.
There are four different types of clinical trials, each one associated with a different phase in the development of a new medicine or treatment. These four phases are:
These trials involve the first administration of the medicine to humans and determine the safety of the medicine, how it works and how well it is tolerated as well as determining the appropriate dose of administration. Phase I trials run for children are almost always run after successful Phase I studies in adults have been completed.
These trials are run after the safety and correct dose has been determined in Phase I studies. The main aim of these trials is to determine the effectiveness of the new medicine or treatment and trials are undertaken in a small number of closely supervised patients.
These trials involve greater numbers of patients and are undertaken to determine whether the medicine or treatment is more effective than the current standard treatment used for a particular disease. Phase III trials are undertaken once the Phase II trials have determined that the medicine has potential benefits that outweigh any hazards experienced with the new medicine or treatment.
Phase IV trials are those undertaken after the medicine has been approved for the treatment of a particular disease and are used to compare the new medicine with a wider range of existing medicines/therapies.
Participation in a clinical trial is voluntary and once you have agreed to your child’s participation you are always able to change your mind and withdraw your child from the trial. Sometimes participation in a clinical trial will provide benefits to the participant such as access to services/treatments that are not otherwise freely available and in other cases there may be no direct benefit to the participant or it may be difficult to say whether a participant will receive any benefit. Sometimes there will be benefits in the future for people who are diagnosed with cancer. It is also possible that a trial is not appropriate for a particular child, each situation is considered individually. All this will be explained to you by the medical staff who will talk to you about the details of the clinical trial that your child is being asked to participate in.