7/10/2018 Update – Trial is now recruiting ages 2-7 years old, previously it was recruiting 32-66 months.
Enabling Future Clinical Trials: Noninvasive EEG to Find Neural Markers of Fragile X
Dr. Carol Wilkinson, MD PhD, and Dr. Charles Nelson, PhD, at the Labs of Cognitive Neuroscience at Boston Children’s Hospital are recruiting young boys (2-7 years) with Fragile X syndrome (FXS) to participate in a study investigating how differences in brain activity affect learning, language, and behavior in FXS.
For more information or to enroll in this study, please contact Dr. Carol Wilkinson at FXSNeuralMarkers@childrens.harvard.edu or (617) 355-4373.
Biomarkers are objective measures that can be used to determine whether – and how well – a treatment is working. There is an enormous need for good biomarkers for treatment trials in Fragile X syndrome. This study will test whether electroencephalography (EEG) can identify useful neural markers (biomarkers for the brain).
EEG is a simple, non-invasive technique that can identify differences in the patterns of brain activity. EEG-based neural markers may also predict specific challenges, like speech delay or behavioral problems. Once neural markers are found for particular challenges, such as kids with poor language versus good language, those markers can be measured during drug and behavioral therapy trials to see if a child is improving based on objective biological readouts. This would be enormously helpful in determining whether or not a treatment is truly working.
What is Involved in Participating?
Young boys with Fragile X between the ages of 2-7 years may participate. A single 4 hour visit to Boston Children’s Hospital will be scheduled at the family’s convenience, including weekends, and siblings will be allowed to accompany the family. This visit includes clinical and behavioral assessment that can evaluate a child’s thinking, motor skills, language skills, and social communication. In addition the study will measure participant’s brain activity using electroencephalography (EEG).
EEG is a safe and radiation free way to measure brain activity in response to different sounds and pictures. The EEG evaluation rooms, themed Jungle, Mario, Aquarium and Farm, are quiet, with comfy couches in the outer rooms. In Wilkinson’s studies, there is an EEG cap, like a hair net with imbedded buttons and a chin strap to hold it on snugly. The child sits on a parent’s lap on a chair and is assisted by a research assistant while another researcher collects the data.
Families will receive a small toy and $25 for participating. Free parking and child care for siblings are also provided.
During the EEG recordings, Wilkinson breaks stimuli down into simple tasks, looking at brain responses to visual and auditory tests compared to a resting situation. The visual tests involve looking at checkerboards with reversing patterns, and the auditory stimuli are beeps with varying frequencies, very similar to hearing tests. This is why the EEG room looks so much like an audiology lab. While the children look and listen, data is collected.
Benefits of this Study
The study provides no benefit to the children who participate, but if it results in reliable neural markers, the Fragile X field will have a powerful and safe tool to assess treatments in upcoming clinical trials. Wilkinson says “given that a large number of children with Fragile X have autism, we hope that some of the same neural markers we find in Fragile X will also translate to a subset of children with autism. If there are therapies that work in Fragile X, then we could identify kids with autism or other neurological disorders with a similar EEG profile who could be helped by the same therapies or drugs.”
“In mouse models of Fragile X many drugs show promise but then have failed in clinical trials. This is not necessarily because they don’t work well, but rather that they may only be effective in a subset of the population, and then don’t demonstrate a positive outcome when averaged together,” explains Wilkinson. If we can determine what distinguishes one brain from another, and if a drug works with a particular neural marker or set of neural markers, this would permit matching drugs based on objective biological markers, a personalized medicine approach, rather than defaulting to the current method of trial and error.
Additional Insight into this Clinical Study of EEG
Last year we published Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials which gives further insight into this clinical study and the benefits from it.
If you are interested in this study, Dr. Carol Wilkinson has created a social story to help explain it to your child.
For more information or to enroll in the study, please contact Dr. Carol Wilkinson at FXSNeuralMarkers@childrens.harvard.edu or (617) 355-4373