UCL Queen Square Institute of Neurology


Ataxia Centre research into attitudes of people with ataxia towards clinical trials

4 March 2022

Researchers at Ataxia Centre, UCL Queen Square Institute of Neurology, UCLH, Children’s Hospital of Philadelphia, Ataxia UK and Friedreich’s Ataxia Research Alliance (FARA) have published research looking at the attitudes of people with ataxia towards clinical trials.

Meeting of the Volunteers of the Ataxia Centre

The team wanted to understand the motivations for and the barriers to clinical trial participation. This information is important when designing future clinical trials to improve recruitment, participation, and trial experience for people with ataxia.

To collect this information, Ataxia UK and FARA sent a survey to their members. This included questions on demographics, personal motivation, drug therapy, and trial design. We would like to thank all those who took part! In total, 342 people responded to the survey, including 204 people with Friedreich’s ataxia (FA), 55 people with inherited cerebellar ataxia (CA), and 70 people with idiopathic CA (CA with no known cause).

The results showed that the most important symptoms that non-wheelchair users wanted to address in a trial were balance problems and walking. For wheelchair users, the most important symptom was speech problems. In both groups, fatigue was also an important symptom to be addressed.

The most common motivation for people to participate in a trial was the potential benefit to themselves and others, while the most common reasons for people not to participate were fear of side effects and the burden and cost of travel. Financial reimbursement for trial participants was reported to likely increase engagement in trials.

Overall, people were most willing to participate in Phase 2 trials, which look at how well a treatment works and its side effects. Survey respondents reported that potentially being given a placebo in a trial was a disincentive, although more people would be willing to participate if an open-label extension was offered. An open-label extension is when trial participants who are given a placebo in an initial trial are then invited to take the study drug once the initial trial has been completed. Drug repurposing trials were also popular among respondents.

Although most medical procedures were deemed acceptable by most respondents, more invasive procedures were less accepted. Encouragingly, 70% of people were prepared to do a lumbar puncture in order to administer a drug intrathecally (ie: injection into the spinal fluid) as part of a trial.

Project leader, Prof Giunti, says: “Patient recruitment and retention are vital to the success of clinical trials. We therefore looked at barriers to participation in order to improve the success of clinical trials. The results of our project are relevant to the pharmaceutical industry and academics for trial design, especially in rare diseases.”

Overall, this study informs trial design which is more focused towards people with ataxia and may improve the recruitment and retention of participants to future trials.


Thomas-Black, G., Dumitrascu, A., Garcia-Moreno, H. et al. The attitude of patients with progressive ataxias towards clinical trialsOrphanet J Rare Dis 17, 1 (2022). https://doi.org/10.1186/s13023-021-02091-x