Facilitating individuals and families affected by fragile X syndrome to participate in medication trials.
Fragile X families skip drug trials because they fear side effects and logistics—offer liquid meds, blood-test practice, and remote visits to bring them in.
01Research in Context
What this study did
Mulder et al. (2020) asked 328 fragile X families what keeps them out of drug studies.
They ran surveys and small group talks. Parents, adults with FXS, and doctors all gave views.
What they found
Biggest fear: side effects. Next headache: travel, blood draws, and missed work.
Cheap fixes help. Offer liquid medicine, practice blood tests at home, and let visits be remote.
How this fits with other research
Robertson et al. (2013) showed therapy use drops sharply after age twenty. The new data say families still want care, just not trials that add stress.
Waldron et al. (2023) found Medicaid families loved COVID-era tele-health. The FXS study echoes that: remote visits could raise study sign-ups.
Matson et al. (1994) taught parents to coach kids through spinal taps. The same coach-and-practice plan fits blood-test fears in FXS trials.
Why it matters
You can ease families into research today. Swap pills for liquid, mail a practice kit, hold the first visit on Zoom. Small moves may fill seats in future fragile X drug studies.
Want CEUs on This Topic?
The ABA Clubhouse has 60+ free CEUs — live every Wednesday. Ethics, supervision & clinical topics.
Join Free →Send a parent a short video that shows how to rehearse blood draws at home before the first trial visit.
02At a glance
03Original abstract
BACKGROUND: Recently, there has been an increasing number of trials of medications for fragile X syndrome (FXS). In order to be adequately powered, trials have involved many centres around the world with relatively small numbers of participants recruited at each site. This study aims to understand the barriers to, and how best to facilitate participation in, medication trials in order to improve recruitment and the experience of participants with FXS. METHODS: A mixed methods design was used to collect both quantitative and qualitative data. Participants were invited to participate through the UK Fragile X Society, a local mailing list and through social media. Those who agreed to participate completed a quantitative questionnaire and indicated whether they would be willing to participate in a follow-up focus group. RESULTS: The questionnaire was completed by 328 individuals who either had FXS, or were a parent, carer or family member of an individual with FXS. Over two-thirds of participants reported concern about side effects, while over one-third mentioned swallowing tablets, blood tests, financial aspects and travel as barriers to participation. Focus groups with 12 individuals highlighted themes of trial challenges, strategies to overcome these and motivating factors to participate. CONCLUSIONS: Many of the factors, which potentially negatively influence participation in a clinical trial for FXS, could be mitigated in relatively simple ways. Easily accessible information, particularly about safety issues, the research team and the trial environment should be standard practice. Desensitisation programmes for blood testing, provision of different preparations of medication (e.g. liquid) and use of a combination of local, remote and site visits to reduce travel and time should also be considered.
Journal of intellectual disability research : JIDR, 2020 · doi:10.1111/jir.12779