Your browser doesn't support javascript.
loading
New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures.
Boxer, Adam L; Gold, Michael; Feldman, Howard; Boeve, Bradley F; Dickinson, Susan L-J; Fillit, Howard; Ho, Carole; Paul, Robert; Pearlman, Rodney; Sutherland, Margaret; Verma, Ajay; Arneric, Stephen P; Alexander, Brian M; Dickerson, Bradford C; Dorsey, Earl Ray; Grossman, Murray; Huey, Edward D; Irizarry, Michael C; Marks, William J; Masellis, Mario; McFarland, Frances; Niehoff, Debra; Onyike, Chiadi U; Paganoni, Sabrina; Panzara, Michael A; Rockwood, Kenneth; Rohrer, Jonathan D; Rosen, Howard; Schuck, Robert N; Soares, Holly D; Tatton, Nadine.
Afiliación
  • 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.
  • McFarland F; Department of Medicine (Neurology), Sunnybrook Health Sciences Centre, University of Toronto, ON, Canada.
  • Niehoff D; McFarland Writing, Annapolis, MD, USA.
  • Onyike CU; Association for Frontotemporal Degeneration, Radnor, PA, USA.
  • Paganoni S; Department Geriatric Psychiatry and Neuropsychiatry, Johns Hopkins University, Baltimore, MD, USA.
  • Panzara MA; Healey Center for ALS, Massachusetts General Hospital, Boston, MA, USA.
  • Rockwood K; Development, Wave Life Sciences, Boston, MA, USA.
  • Rohrer JD; Division of Geriatric Medicine, Dalhousie University, Halifax, NS, Canada.
  • Rosen H; Dementia Research Centre, UCL Institute of Neurology, Queen Square, London, UK.
  • Schuck RN; Memory and Aging Center, Department of Neurology, University of California San Francisco, San Francisco, CA, USA.
  • Soares HD; Office of Clinical Pharmacology, Center for Drug Evaluation and Research, FDA, Silver Spring, MD, USA.
  • Tatton N; Department of Neurology, AbbVie, Chicago, IL, USA.
Alzheimers Dement ; 16(1): 131-143, 2020 01.
Article en En | MEDLINE | ID: mdl-31668596
ABSTRACT

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.
Asunto(s)
Palabras clave
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Imagen por Resonancia Magnética / Biomarcadores / Ensayos Clínicos como Asunto / Degeneración Lobar Frontotemporal Límite: Humans Idioma: En Revista: Alzheimers Dement Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Imagen por Resonancia Magnética / Biomarcadores / Ensayos Clínicos como Asunto / Degeneración Lobar Frontotemporal Límite: Humans Idioma: En Revista: Alzheimers Dement Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos