What we offer
The Centre for Clinical Trials in Rare Neurodevelopmental Disorders offers new treatment opportunities for your child that may not currently be widely available as a typical treatment avenue.
What is a clinical trial?
A clinical trial develops a safe and controlled environment to test new treatments and provide evidence as to how new treatments or interventions might work to improve health conditions and services. Research involving children, from babies to adolescents, is essential to improve our understanding of childhood diseases and conditions and provide care for children and young people based on the best possible evidence. At present, many medicines given to children have not been tested specifically with children. As a result, there is only limited evidence available to prescribers about the most appropriate dosage, and about how children may respond¹.
New drug treatments are tested to ensure their safety by going through phased trials.
- Phase 1: clinical safety trials to identify dosage range and side effects;
- Phase 2: efficacy and safety trials in a larger group (several hundred);
- Phase 3: clinical trial in a large group to monitor efficacy and side effects;
- Phase 4: studies to monitor drug therapy effectiveness in the population.
The findings of clinical trials are collated and made available through a variety of channels including scientific publications. The ‘levels of evidence pyramid’ demonstrates how clinical trials contribute to evidence-based decisions about clinical treatment options for our patients.
Figure 1: Evidence-based medicine pyramid. Sackett, D. et al, 2000
How can a clinical trial benefit you?
A clinical trial may allow your child to access new treatments that are not available on the general market. Taking part in a clinical trial may or may not benefit you or your child directly. In some circumstances it may be helpful to determine if a particular medication might be suitable for your child such as in cross-over trials. However, participation in trials will further advance information as to the best solutions to support children with a variety of neurodevelopmental challenges, helping other families and children across the globe.
What do I do to get involved in a clinical trial?
There are certain criteria that a clinical trial need to meet to proceed safely and within a research code of ethics. You can express your interest in a trial by providing basic demographic information for your child like age and gender and any specific information such as current medications or medical diagnosis. You may be accepted to join the trial and be ‘screened’ to ensure your child will be suitable for the trial and that the trial may benefit them.
What is a rare neurodevelopmental disorder?
The term neurodevelopmental disorder is used in two ways, firstly to describe genetic conditions which affect a child’s neurological development such as Fragile X Syndrome. Secondly, to highlight conditions of multi-faceted origin which impair neurodevelopment such as autism spectrum disorders2. A rare neurodevelopmental disorder refers to one which is not commonly seen and affects a small number of people. The Centre for Clinical Trials in Rare Neurodevelopmental Disorders supports research that could provide new treatment and interventions to improve the health conditions and services available to this population.
2Bishop DV. Which neurodevelopmental disorders get researched and why?. PLoS One. 2010;5(11):e15112. Published 2010 Nov 30. doi:10.1371/journal.pone.0015112