The longitudinal evaluation of familial frontotemporal dementia subjects protocol : framework and methodology

Introduction It is important to establish the natural history of familial frontotemporal lobar degeneration (f‐FTLD) and provide clinical and biomarker data for planning these studies, particularly in the asymptomatic phase. Methods The Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects protocol was designed to enroll and follow at least 300 subjects for more than at least three annual visits who are members of kindreds with a mutation in one of the three most common f‐FTLD genes—microtubule‐associated protein tau, progranulin, or chromosome 9 open reading frame 72. Results We present the theoretical considerations of f‐FTLD and the aims/objectives of this protocol. We also describe the design and methodology for evaluating and rating subjects, in which detailed clinical and neuropsychological assessments are performed, biofluid samples are collected, and magnetic resonance imaging scans are performed using a standard protocol. Discussion These data and samples, which are available to interested investigators worldwide, will facilitate planning for upcoming disease‐modifying therapeutic trials in f‐FTLD.

Language

English

Source (journal)

Alzheimer's & dementia / Alzheimer’s Association [Chicago, Ill.] - Orlando, Fla, 2005, currens

Publication

Orlando, Fla : Elsevier , 2020

ISSN

1552-5260 [print]

1552-5279 [online]

DOI

10.1016/J.JALZ.2019.06.4947

Volume/pages