Paper published on the attitudes of people with ataxia towards clinical trials - Ataxia UK

Paper published on the attitudes of people with ataxia towards clinical trials

Post Published: March 4, 2022

In January 2022, researchers at UCL/UCLH, Children’s Hospital of Philadelphia, Ataxia UK and Friedreich’s Ataxia Research Alliance (FARA), published a paper on the attitudes of people with ataxia towards clinical trials.

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

Ataxia UK and FARA sent a survey to their members, including questions on demographics, personal motivation, drug therapy and trial design. Thank you to all those who took part! 342 people responded, 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 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. 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 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. 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. This 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.”

The full paper can be found at 


Date posted: 04/03/2022

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