A new study by the UC Davis MIND Institute’s David Hessl will test whether a smartphone-based app may help improve the accuracy of data in some clinical trials involving individuals with intellectual disabilities. ‘It’s a brilliant use of a ubiquitous technology, and it could make a huge difference in the accuracy of measurement in clinical trials,’ said Hessl, a professor in the Department of Psychiatry and Behavioral Sciences.
Hessl was awarded a two-year, $431,000 grant from the National Institutes of Health (NIH) for the project. It’s part of the NIH INCLUDE initiative centred on investigating conditions that co-occur with Down syndrome across the lifespan, including ADHD.
Hessl’s study will focus on children and young adults with intellectual disabilities. The most common genetic causes of intellectual disability are Down syndrome and fragile X syndrome. The goal is to track symptoms related to executive function, often associated with ADHD, using the new smartphone app called iBehavior.
A quest for more accurate data
“The majority of clinical trials focused on people with intellectual disability have relied on parent or caregiver questionnaires, much like traditional paper and pencil rating scales,” explained Hessl.
That means families have to remember over a reasonably long period – sometimes weeks – what their child’s behaviour was like.
‘The app uses what we call a momentary ecological assessment, where a parent or teacher observes the child or young adult and provides ratings of behaviour much closer to the time the behaviour occurred,’ said Hessl. ‘People have varying degrees of accuracy with their memory, and we are all subject to different kinds of bias, which can negatively impact studies. So, we’re hoping, with the more immediate recording of the information, we’ll get a more sensitive and accurate representation of whether patients are improving or not.’
Families will be trained in how to use iBehavior and what behaviour to look for. The app will send a text when it’s time for them to start observing their child or young adult; they’ll observe for two hours, then get another text that it’s time to record what they observed. They’ll record information about aggression and irritability, hyperactivity and impulsivity, attention problems, anxiety-related behaviour and avoidance and more, likely daily. Classroom teachers may also be asked to take part.
The new app was Hessl’s idea, and Michael Bass of Northwestern University programmed iBehavior with guidance from Hessl’s team and a group of consultants.
Building on existing research
Researchers will evaluate the tool with families who are taking part in another NIH-funded long-term study called “A Cognitive Test Battery for intellectual disabilities. Hessl is also the Principal Investigator of this study. ‘We are working to leverage all of what we’ve learned in this parent study by pairing up with the iBehavior app method,’ he said.
As part of the parent project, Hessl and his colleagues recently validated the NIH Toolbox Cognition Battery, a series of tests delivered on a tablet or iPad to assess cognitive function for use in individuals with intellectual disabilities.
‘We’ll invite families to provide data on the app, as well as take part in the traditional assessments and that way, and we’ll be able to cross-compare. For example, we’d expect that if a parent reports a high level of executive function difficulties in the child’s daily life, for example, impulsivity or inattention, that the Toolbox, which measures cognition in the lab, would show those same kinds of difficulties,’ Hessl explained.
A family’s gift enables a new approach to research
Before Hessl could apply for NIH funding, his idea had to be developed. That’s where Brian and Shari Silver come in. Their son, Justin, has fragile x syndrome and has received treatment and participated in research studies at the MIND Institute. They believed in iBehavior and made donations, both personally and through their non-profit organisation, the Fly With Me Fund, critical to the app’s development.
‘When the opportunity was presented to support Dr Hessl in getting a new project off the ground, there was no doubt we would do what we could,’ said Shari Silver. ‘Having participated in the research, and also having had to evaluate medications as they were introduced to our son, we recognised that if we had a tool like iBehavior, capturing our observations daily with the battery of questions in the app would have helped us to provide the most accurate feedback to clinicians.’
There is currently no treatment for fragile X syndrome; there are only treatments for symptoms. Silver noted that they are not always effective, and she’s hopeful that this app will improve the assessment of a child’s behaviour before, during and after treatment and improve the evaluation of potential targeted therapies.
‘This is a game-changer for our son’s future and all families living with fragile X syndrome and other neurodevelopmental conditions,’ she said. The Silvers have supported other MIND Institute research, including a new blood processing machine for laboratory work, travel grants for patients with fragile x syndrome to visit the MIND Institute and telehealth services. They’ve also tested the iBehavior app, and Shari is sure of its viability.
What’s next for iBehavior
Starting in 2023, the study will also include a clinical trial of stimulant medication, a standard treatment for ADHD.
Hessl notes that several clinical trials have been negative – or showed medications to be ineffective. There is debate about whether some treatments are genuinely ineffective or whether the previously used outcome measures were adequately sensitive to patient behavioural change.
‘For example, if a trial fails, we assume it’s because the medication was not effective, but there’s a possibility that there was some efficacy, but the tools used weren’t sharp enough, not accurate enough to pick up the improvement.’ The hope is to validate the use of the app as an outcome measure for clinical trials.