Feasibility of clinical research studies reported by patients with behavioral variant frontotemporal dementia and their caregivers

Background Behavioral variant frontotemporal dementia (bvFTD) is a neurodegenerative disorder characterized by progressive, profound behavioral changes plus loss of insight and independence, leading to significant burden for affected families both in daily life and clinical research participation. FORWARD study aimed to estimate disease‐related decline in patients with early‐stage bvFTD (NCT03088956) over 1 year (consisting of 6 in‐clinic and at least 4 at home study visits), using a battery of cognitive tests, functional, behavioral, and quality of life assessments. Participants were asked to complete an end‐of‐study questionnaire with the purpose to identify aspects of the study considered as potentially burdensome, challenging and/or positive. Method The one‐time end‐of‐study questionnaire consisting of 10 questions was completed by 29 bvFTD subjects and their 29 caregivers at their last study visit (Month 13 or Early Termination). Result Majority of participants were very satisfied in taking part in the study (66% bvFTD; 72% caregivers). The top motivators to participation included contributing to dementia research (79% bvFTD; 93% caregivers) while the main drivers to complete the study included their commitment to be in the study (83% bvFTD; 86% caregivers) as well as the amount of care and attention received from the study doctor and staff (76% bvFTD; 79% caregivers). In terms of study design, home visits were considered useful (76% bvFTD; 83% caregivers) and not burdensome (79% bvFTD; 86% caregivers) by the majority of participants. Most participants did not find every 3‐months clinic visits (55% bvFTD; 62% caregivers), clinic visit length (62% bvFTD; 76% caregivers), and study duration (59% bvFTD; 72% caregivers) as burdensome. However, bvFTD patients considered burdensome cognitive assessments done either in paper forms (55%) or on iPAD (55%). Use of iPAD was considered useful (48% bvFTD; 64% caregivers) and not burdensome (69% bvFTD; 93% caregivers) mainly by caregivers and less so by patients. Financial travel support was deemed useful (69% bvFTD; 66% caregivers) and 62% of caregivers found the overall study experience not burdensome for bvFTD patients. Conclusion These insights are useful to minimize the degree of burden for participants with improved clinical trial design in the bvFTD indication with limited drug development precedence.