What are they and how can they be addressed by researchers of medication trials for Fragile X Syndrome?
People with Fragile X Syndrome (FXS) are primarily characterised with intellectual disability (ID), and many will have autistic traits as well as symptoms of anxiety, hyperactivity, and hypersensitivity. These aspects of FXS make it particularly difficult for these individuals to consent to and participate in clinical trials. The prevalence of clinical trials is, however, growing in response to high demands for medication which targets core features of FXS instead of just its symptoms. Such features include chemical and functional changes within neurons in the brain and wider nervous system.
To collect opinions on clinical trials, explore barriers to participation, and identify strategies to improve enrolment, parents, carers, or family members of people with FXS and those with FXS themselves were invited to take part in a participatory enhancement study carried out by researchers at the Patrick Wild Centre. A total of 328 questionnaires were completed by participants from 13 countries around the world, and 12 of these participants partook in focus groups.
Questionnaire data revealed concerns over side-effects to be the most prominent barrier to participation in clinical trials. To address this, it was suggested that trials offer an abundance of safety data in an accessible, easily interpreted format. This would help individuals with FXS and their families to make informed decisions about participation in full awareness of the risks involved. Hosting information evenings to meet the research team, ask questions, and visit the testing facilities was another suggestion to build trust and diminish concern. Demands of travel, financial strain, and timing were other commonly expressed barriers. Reducing the number of visits to distant locations, maintaining flexibility with local and/or remote testing, and where possible organising shared transport were insightful recommendations to overcome these practical barriers and create support systems between participants. Finally, discomfort of taking blood and swallowing pills were procedural barriers which surfaced in the data. Providing information or techniques to desensitise the participants with FXS to blood testing, as well as offering alternative forms of administering medication (injection, patches, gels, etc.) were suggested to address these procedural difficulties. Researchers importantly highlighted that these barriers are applicable not only to clinical trials for individuals with FXS, but also to those for individuals with idiopathic ID and autism.
Equipped with an awareness of these barriers and strategies to overcome them, research teams can make important modifications to recruitment and testing protocols in a way that optimises clinical trial design, encourages widespread participation, and enhances validity of scientific results. Such improvements are essential to mobilise effective investigations and test new medicines for FXS.
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Emma Mather-Pike
Research Information Manager
Fragile X Society
24 March 2022