New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures.

Autor:	Boxer AL; Memory and Aging Center, Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Gold M; Development Neurosciences, AbbVie, Chicago, IL, USA., Feldman H; Department of Neurosciences, University of California San Diego, San Diego, CA, USA., Boeve BF; Department of Neurology, Mayo Clinic, Rochester, MN, USA., Dickinson SL; Association for Frontotemporal Degeneration, Radnor, PA, USA., Fillit H; Alzheimer's Drug Discovery Foundation, New York, NY, USA., Ho C; Denali Therapeutics, San Francisco, CA, USA., Paul R; Alector, Inc., South San Francisco, CA, USA., Pearlman R; The Bluefield Project, San Francisco, CA, USA., Sutherland M; Chan Zuckerberg Initiative, Redwood City, CA, USA., Verma A; United Neuroscience, Dublin, Ireland., Arneric SP; Critical Path Institute, Tucson, AZ, USA., Alexander BM; Dana-Farber Cancer Institute, Harvard University, Boston, MA, USA., Dickerson BC; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA., Dorsey ER; Center for Health and Technology, University of Rochester, Rochester, NY, USA., Grossman M; Department of Neurology, University of Pennsylvania, Philadelphia, PA, USA., Huey ED; Departments of Psychiatry and Neurology, Columbia University, NY, USA., Irizarry MC; Formerly of Early Phase Neurosciences, Eli Lilly, Indianapolis, IN, USA., Marks WJ; Clinical Neurology, Verily Life Sciences, South San Francisco, CA, USA., Masellis M; Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, University of Toronto, ON, Canada.; Department of Medicine (Neurology), Sunnybrook Health Sciences Centre, University of Toronto, ON, Canada., McFarland F; McFarland Writing, Annapolis, MD, USA., Niehoff D; Association for Frontotemporal Degeneration, Radnor, PA, USA., Onyike CU; Department Geriatric Psychiatry and Neuropsychiatry, Johns Hopkins University, Baltimore, MD, USA., Paganoni S; Healey Center for ALS, Massachusetts General Hospital, Boston, MA, USA., Panzara MA; Development, Wave Life Sciences, Boston, MA, USA., Rockwood K; Division of Geriatric Medicine, Dalhousie University, Halifax, NS, Canada., Rohrer JD; Dementia Research Centre, UCL Institute of Neurology, Queen Square, London, UK., Rosen H; Memory and Aging Center, Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Schuck RN; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, FDA, Silver Spring, MD, USA., Soares HD; Department of Neurology, AbbVie, Chicago, IL, USA., Tatton N; Association for Frontotemporal Degeneration, Radnor, PA, USA.
Jazyk:	angličtina
Zdroj:	Alzheimer's & dementia : the journal of the Alzheimer's Association [Alzheimers Dement] 2020 Jan; Vol. 16 (1), pp. 131-143. Date of Electronic Publication: 2020 Jan 06.
DOI:	10.1016/j.jalz.2019.06.4956
Abstrakt:	Introduction: Frontotemporal lobar degeneration (FTLD) is the most common form of dementia for those under 60 years of age. Increasing numbers of therapeutics targeting FTLD syndromes are being developed. Methods: In March 2018, the Association for Frontotemporal Degeneration convened the Frontotemporal Degeneration Study Group meeting in Washington, DC, to discuss advances in the clinical science of FTLD. Results: Challenges exist for conducting clinical trials in FTLD. Two of the greatest challenges are (1) the heterogeneity of FTLD syndromes leading to difficulties in efficiently measuring treatment effects and (2) the rarity of FTLD disorders leading to recruitment challenges. Discussion: New personalized endpoints that are clinically meaningful to individuals and their families should be developed. Personalized approaches to analyzing MRI data, development of new fluid biomarkers and wearable technologies will help to improve the power to detect treatment effects in FTLD clinical trials and enable new, clinical trial designs, possibly leveraged from the experience of oncology trials. A computational visualization and analysis platform that can support novel analyses of combined clinical, genetic, imaging, biomarker data with other novel modalities will be critical to the success of these endeavors. (© 2019 the Alzheimer's Association.)
Databáze:	MEDLINE
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