New Directions in Clinical Trials for Frontotemporal Lobar Degeneration: Methods and Outcome Measures

The rarity of disorders caused by frontotemporal lobar degeneration (FTLD) leads to recruitment challenges for related clinical trials and difficulties with adequate power to detect treatment effects. The authors of this systematic review recommend developing personalized endpoints that are most clinically meaningful to individuals and their families, devising more powerful approaches to analyzing heterogeneous clinical and magnetic resonance imaging data, and developing new fluid biomarkers and wearable technologies. These strategies would help to improve the power to detect treatment effects in FTLD clinical trials and enable new, more efficient clinical trial designs modeled on oncology. The authors note that more widespread sharing of clinical trial data and biofluid samples will be critical to developing new endpoints and refining FTLD clinical trial designs.

Boxer AL, Gold M, Feldman H, et al. New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures. Alzheimer’s & Dementia 2020;16(1):131-143.