This is a collection of completed studies where we introduced something new to the participants of the study and then looked to see what the impact of this was. In some cases, this was starting a new medication; in other cases it was using a new application; and in some cases it was attending a training course.
Novartis AFQ056 clinical trials for fragile X syndrome
The results from these trials are negative, i.e. AFQ056 was not found to be effective for the treatment of difficult behaviours in fragile X syndrome. More information is given below.
Results of the Randomised Controlled Trials
Novartis were running two trials to see if their new medicine, AFQ056, was helpful for difficult behaviours in fragile X syndrome; one trial was in adults, the other in adolescents (12 – 17 year olds). Both lasted for 4 months and the aim was to see if AFQ056 was more helpful than a placebo. In these studies, some volunteers were given the active medicine, AFQ056, while the rest were given placebo. During the trial neither the volunteers nor the doctors prescribing the medicine knew whether they were given placebo or AFQ056.
Unfortunately the results from both trials were negative, in other words AFQ056 was no more helpful than placebo. The researchers found that almost everyone in the studies felt they had benefitted in some way regardless of whether they had been given AFQ056 or placebo. The conclusions from these trials are that AFQ056 does not work in any useful way for people with fragile X syndrome.
Novartis also wondered whether they simply had not measured the correct behaviours to show an improvement – if you don’t measure the right things then how can you tell if they are better? So they asked researchers to write a description of how each volunteer improved then looked to see whether certain types of improvements were more common in those on AFQ056 or placebo. They did not find any difference between the two meaning that the lack of benefit was not just because they did not look in the right place.
Finally, they checked to see whether there was any evidence that AFQ056 worked for younger people in the trials and not for older people. There was no evidence that this was the case.
Results of the continuation trials
Those people who were in the 4 month trials described above were offered the chance to stay on the medicine; some people have therefore been taking AFQ056 for more than 2 years. These continuation studies do not have a placebo – i.e. everyone is taking AFQ056. Almost everyone in these studies reported some improvement; however these improvements were no larger than those seen in people taking placebo in the randomized trials. The conclusions from these continuation studies is that even if taken for a long time AFQ056 does not lead to more improvements than would be seen with a placebo.
The Future for AFQ056
As a result of these studies, Novartis have discontinued their research into AFQ056 for fragile X syndrome. The continuation studies are in the process of stopping and the medicine will become unavailable by summer 2014. The lack of any evidence that younger people did better means that they have decided that they will not run the trial in 5 – 11 year old children that they had previously talked about doing.
The Future for Research into Medical Treatments for Fragile X Syndrome
There are a number of other medicines that are currently being considered as potentially helpful for people with fragile X syndrome. Some of them affect the brain in a similar way to AFQ056, some of them affect it differently. At least one of these medicines is being tested in children aged 5 years and over in the USA. Although the results of the Novartis trials are very disappointing the work towards finding medicines that help people with fragile X will continue.
Future Trials in the UK
At the moment we do not have any other trials running in the UK, but the Patrick Wild Centre hopes to run trials of other medicines in the not too distant future. Any research that we do relies upon people volunteering to take part so before we start another trial we are to seeking the views of families about whether this sort of research is important to them and if so how we could go about maximising the chance of success. Take part in our survey on the barriers to fragile X clinical trials here.
Click-East iPad App Study
The Click-East project was divided into two main stages. The first stage was about developing an educational iPad app for young children with autism, and the second stage tested whether it is a successful learning aid. This second stage was a rigorously-designed randomised controlled trial, registered on both the UK Clinical Research Network study portfolio and on the National Institute of Health clinical trials register.
Stage One: completed in March 2012
The first stage of the project involved a range of different people who contributed to the development of the game in different ways. At the University of Edinburgh the project received contributions from computer programmers and human-computer interaction specialists in the School of Informatics and from animators at the Edinburgh College of Art, as well as Dr Sue Fletcher-Watson, a developmental psychologist based at The patrick Wild Centre.
We collected a lot of feedback on the game from typically-developing children, parents of children with autism spectrum disorders, teachers and nursery nurses providing specialist provision for children with autism spectrum disorders, speech and language therapists and young adults with an autism spectrum diagnosis.
Stage Two: completed in June 2013, data analysis ongoing
In Stage Two we tested whether the app we designed had a beneficial effect for children with autism. It is our hope that by learning and practising basic social and communication skills in a safe and fun way, children will start to show these skills in real life too. Using a computer game as a learning tool plays to the strengths of children with autism, who often show a strong preference for technology and a lot of ability in this area.
Data collection was completed in June 2013 and now we’re analysing the information and beginning to write it up for publication. You can read an accessible summary of our preliminary findings in this September 2013 newsletter.
Commercial Partnership: Interface 3
Interface 3 is our commercial partner. To give the app a life beyond the research project (both in terms of lifetime and quality of life) we have engaged in a partnership with this award-winning Edinburgh based company. For more about this decision, have a look at this post on the Blog page. Alongside the research project detailed above, Interface 3 have published the app for worldwide consumption – it is called FindMe and available on iTunes for iPhone and iPad in both Lite and Pro versions.
If you have already purchased the app, we’d love to hear your feedback. Please follow this link to complete a short survey. Your responses will be incredibly valuable in helping us to continue our work on this app, and in proposed future research continuing to explore modern technology-based education and therapy for people with autism spectrum disorders.
East Park iPad App Study
DART researchers spent the last year working with East Park – an organisation in Glasgow providing education, care and support to pupils with additional support needs including autism. The school were fortunate enough to secure funding to buy a number of iPads which were given to selected students and used to support their personal learning goals over the academic year. DART Masters student, Sinead O’Brien, provided an independent evaluation of the project. In this simple partnership we had a number of exciting, applied research goals:
- to evaluate the impact of iPads on individual learning goals and on the classroom setting. We’re fascinated to see how teachers can combine their personal insight into their pupils’ needs and interests, with the wealth of opportunities encapsulated in an iPad, and direct these to impact on learning outcomes in both academic and non-academic domains.
- to consider how iPads can best be implemented in a group classroom setting. As personal technologies become more and more common in classrooms it will be a challenge for teachers to develop ways to respond to them and to ensure their use is effective and relevant to the curriculum and/or personal learning goals. We hope to provide a structure to help teachers reflect on and develop this practice.
- to explore how to evaluate individual iPad use in a school setting – what measures should we be taking and how often? Research in this field requires better measures which can be replicated across studies, settings and specific technologies.
As a result of the project Sinead produced a Masters thesis, which we hope will provide a foundation for further research at PhD level into the educational value of technology for children and young people with autism. We have now also created a report for East Park summarising our independent evaluation of their project. We hope that the school will be able to use this partnership to secure further funding for iPads and other technologies for use in their schools. We are also going to developed guidelines for other schools, to be shared on the Autism Toolbox. Finally, we intend to publish the research in an academic journal for consideration by the research community.
We’re excited about the possibility that the team of East Park plus DART are helping to forge new understanding of the use of tech in schools and can later help to share this knowledge with others. If your school or service is looking to move into using iPads, other tablets or technology for the first time, or you want to enhance this aspect of what you do, please feel free to get in touch.
A social skills intervention for adults with Asperger Syndrome
This study considered whether it was possible to teach adults with Asperger Syndrome techniques to help them understand social situations and hence improve their quality of life. Although such interventions are commonly used in children there are few studies which have considered whether they work in adults. People taking part in this study were randomly allocated to receive one of two group interventions for 16 weeks. The first intervention was a group based around teaching social skills techniques, with a focus initially on fundamental skills followed by a period considered their application to various social situations. The second intervention was designed to act as a comparison group, with participants attending a group to encourage social interaction, without any particular focus on consciously teaching social skills. The purpose of the second group was to find out whether any improvements seen in the first group related to the specific teaching of social skills or whether they were due to non-specific improvements resulting from being in a group situation or research study. The results of this study are currently being prepared for submission to a research journal and will be posted here when they are available. You can see a summary of the manual used for the social skills intervention here.