Helping people affected by Fragile X Syndrome to participate in medication trials

by: Sarah E.A. Eley; Andrew G. McKechanie; Sonya Campbell; Andrew C. Stanfield

Over the last ten years there have been lots of trials of medications for fragile X syndrome carried out around the world. We wanted to understand what factors affect whether people take part in these trials and what we can do to help those who want to take part overcome any obstacles they may have.

328 people from 13 countries completed a questionnaire which was circulated through the Fragile X Society and online. We then went on to explore the answers from the questionnaire in more detail by carrying out three focus groups around the UK.

Of the 328 people who completed the questionnaire, about a fifth had taken part in a drug treatment trial of a new medicine before and over half of those had had a positive experience, with only 3% of people saying they had had a negative experience. We found that worries about side effects was the main reason why people chose not to take part in clinical trials. Worries over the study procedures and the effects of the study on wider life were also significant reasons why people chose not to take part.

We talked about these concerns in more detail during the focus groups and revealed ways by which they may be overcome. One thing that researchers can do to make things easier is to provide straightforward information to help families understand the study. People thought that having information on any potential safety issues presented in an easy-to-understand format was particularly important. People felt that a good way to get information on the study would be to meet the research team at information evenings and to have an opportunity to ask questions in an informal setting. The families suggested that if questions could be asked at an early stage then this would help to reduce anxieties and promote trust. This means that people would feel more able to take part.

Overall therefore we recommend that, when designing clinical trials for fragile X, researchers should:

1. Provide more information in accessible and easy-to-understand formats, including as much safety data as is possible.

2. Hold information evenings where families can meet the research team and ask questions.

3. Provide materials to help people with FXS familiarise themselves with the research environment and team in advance.

4. Provide medication in different preparations

5. When blood tests are required, provide information to help prepare individuals with FXS for this. If possible, design a desensitisation programme to facilitate blood testing.

6. Consider the number of visits needed for the study and the travel and time that would be required, looking at whether it would be feasible to do a combination of local, remote and more distant site visits if required.

You can find out more about the results from this questionnaire here.

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