TY - JOUR
T1 - Factors impacting trial participation in people with motor neuron disease
AU - Beswick, Emily
AU - Johnson, Micheala
AU - Newton, Judith
AU - Dakin, Rachel
AU - Stenson, Amy
AU - Abrahams, Sharon
AU - Carson, Alan
AU - Chandran, Siddharthan
AU - Pal, Suvankar
N1 - Funding: The authors would like to thank the Euan Macdonald Centre for Motor Neuron Disease Research and the Anne Rowling Regenerative Neurology Clinic for their funding support.
PY - 2023/10/3
Y1 - 2023/10/3
N2 - Motor neuron disease (MND) is a rapidly progressive neurodegenerative disorder with limited treatment options. Historically, neurological trials have been plagued by suboptimal recruitment and high rates of attrition. The Motor Neuron Disease–Systematic Multi-Arm Randomised Adaptive Trial (MND–SMART) seeks to identify effective disease modifying drugs. This study investigates person-specific factors affecting recruitment and retention. Improved understanding of these factors may improve trial protocol design, optimise recruitment and retention. Participants with MND completed questionnaires and this was supplemented with clinical data. 12 months after completing the questionnaires we used MND–SMART recruitment data to establish if members of our cohort engaged with the trial. 120 people with MND completed questionnaires for this study. Mean age at participation was 66 (SD = 9), 14% (n = 17) were categorised as long survivors, with 68% (n = 81) of participants male and 60% (n = 73) had the ALS sub-type. Of the 120 study participants, 50% (n = 60) were randomised into MND–SMART and 78% (n = 94) expressed interest an in participating. After the 1-year follow-up period 65% (n = 39) of the 60 randomised participants remained in MND–SMART. Older age was significantly associated with reduced likelihood of participation (OR = 0.92, 95% CI = 0.88–0.96, p = 0.000488). The findings show that people with MND are highly motivated to engage in research, but older individuals remain significantly less likely to participate. We recommend the inclusion of studies to explore characteristics of prospective and current participants alongside trials.
AB - Motor neuron disease (MND) is a rapidly progressive neurodegenerative disorder with limited treatment options. Historically, neurological trials have been plagued by suboptimal recruitment and high rates of attrition. The Motor Neuron Disease–Systematic Multi-Arm Randomised Adaptive Trial (MND–SMART) seeks to identify effective disease modifying drugs. This study investigates person-specific factors affecting recruitment and retention. Improved understanding of these factors may improve trial protocol design, optimise recruitment and retention. Participants with MND completed questionnaires and this was supplemented with clinical data. 12 months after completing the questionnaires we used MND–SMART recruitment data to establish if members of our cohort engaged with the trial. 120 people with MND completed questionnaires for this study. Mean age at participation was 66 (SD = 9), 14% (n = 17) were categorised as long survivors, with 68% (n = 81) of participants male and 60% (n = 73) had the ALS sub-type. Of the 120 study participants, 50% (n = 60) were randomised into MND–SMART and 78% (n = 94) expressed interest an in participating. After the 1-year follow-up period 65% (n = 39) of the 60 randomised participants remained in MND–SMART. Older age was significantly associated with reduced likelihood of participation (OR = 0.92, 95% CI = 0.88–0.96, p = 0.000488). The findings show that people with MND are highly motivated to engage in research, but older individuals remain significantly less likely to participate. We recommend the inclusion of studies to explore characteristics of prospective and current participants alongside trials.
KW - motor neuron disease
KW - amyotrophic lateral sclerosis
KW - clinical trials
KW - recruitment
U2 - 10.1007/s00415-023-12010-8
DO - 10.1007/s00415-023-12010-8
M3 - Article
SN - 0340-5354
JO - Journal of Neurology
JF - Journal of Neurology
ER -