Clinical Trials
What is a clinical trial?
A clinical trial or study is a medical research study conducted in human subjects to find better ways to prevent, detect, diagnose or treat a disease.
- Which trials? - In our clinical trials for childhood cancer, the existing standard of care treatment is usually compared to a new drug or a slightly adapted treatment to improve the cure rates and/or to reduce relapses and long-term side effects (therapy optimization trials). Belgium is part of a large collaboration between European and/or international study groups and experts joining their knowledge and expertise for the set-up and performance of mostly academic clinical trials. If no clinical trial is available for a certain type of cancer, children and young adults will be treated according to the current standard of care treatment or national/international approved guidelines that are available. Every child will always receive the best available treatment.
- Why? - The number of children and adolescents with cancer is relatively small so participation in large (mostly academic) clinical trials is essential to gather enough data to draw meaningful conclusions regarding better treatment options. Many children and young adults with cancer are treated in large clinical trials since several decades. This has contributed to the better cure rates for children with cancer in general.
What are the trial types?
Academic trials
An academic or non-commercial trial is a clinical trial that is not initiated and funded by a pharmaceutical or biotechnology company for commercial ends. These trials are organized by a public non-profit institution, usually a university or university hospital. The main goal of academic clinical trials is the improvement and optimization of already existing therapies (phase III trials). Most of the trials in children with cancer are large academic international therapy optimization trials in order to improve the existing treatment.
Commercial trials
Commercial trials are run by pharmaceutical companies, which take responsibility to initiate and fund the trial. A large number of new anti-cancer drugs have become available in recent years. Currently, only a few of these new drugs are approved for paediatric use. However, paediatric clinical trials assessing the use and usefulness of these drugs in children, are gradually becoming more frequent.
Trial phases
- Phase I trials are testing new drugs, often for the first time and are helping us to find the correct dose. These trials may look at whether a drug or treatment is safe and/or which side effects it causes. They are performed only in a small group of patients, usually for those patients who received already all possible treatment options in the past (advanced cancer patients).
- Once the correct paediatric dose is found, a phase II trial will test if this treatment is also effective in a larger group of patients, and specifically focuses on the most effective dose and on side effects. A phase II study can also examine if a drug can be given in a different but possibly more effective way. Some phase II trials are randomized, this means that the patients are randomly assigned to one of the different treatment groups.
- In phase III trials, a new treatment will be compared to the existing standard treatment, involving a larger number of patients. These trials are the most common type of trials performed in children with cancer (therapy optimization trials) and they are mostly randomized. These trials can run over a relatively long time period and are typically organised internationally because large patient numbers are needed.
Do I need to participate?
No, participation in a clinical trial is voluntary. Even after you agreed to participate, you are free to withdraw your child from a clinical trial at any moment without having to justify. This will not affect the medical follow-up of your child or the quality of the subsequent care or relationship with your child’s doctor. Your child will always receive the best care and a child’s best interest remains always primordial.
- Informed content - Your child’s doctor will explain the whole trial and its advantages and disadvantages in detail with you and your child. If you decide that your child will participate, you will be asked to sign an informed consent form. This form explains what the trial is about and the doctor will go through the form together with you. You will receive a copy of this signed consent form.
For patients below 18 years, both parents always need to sign the informed consent form. Adolescents from 12 to 17 years also receive a separate information form and need to sign an assent form. Patients from 8 to 11 years old only receive an information form. - Legislation - Conducting a clinical trial requires compliance with a large series of rules and legislations in order to protect the study subject (the patient or the child) and to ensure scientific meaningful outcomes. All the clinical trials that are potentially presented to you, are approved by several ethics committees and/or competent authority and are compliant with all applicable Belgian laws regarding clinical trials and data privacy protection.
Which trials are open?
All doctors in the Belgian centres for paediatric haemato-oncology are familiar with the clinical trials that are open and whether your child is eligible to participate or not. If an appropriate clinical trial is available for the treatment of your child in (one of) the Belgian centres, this will usually be discussed with you by the treating paediatric haemato-oncologist.
If there is no clinical trial open in Belgium that fits your child’s disease, your child will be treated with the current standard of care treatment or according to national/international approved guidelines. Your child will always receive the best available treatment. For more information you can contact your child’s doctor anytime.