Last updated on September 2018

Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)


Brief description of study

Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes: frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is to build a FTLD clinical research consortium to support the development of FTLD therapies for new clinical trials. The consortium, referred to as Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL), will be headquartered at UCSF and will partner with six patient advocacy groups to manage the consortium. Participants will be evaluated at 14 clinical sites throughout North America and a genetics core will genotype all individuals for FTLD associated genes.

Detailed Study Description

Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes: frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is to build a FTLD clinical research consortium (FTLD CRC) to support the development of FTLD therapies for new clinical trials. The FTLD CRC will be headquartered at UCSF and will partner with six patient advocacy groups to manage the consortium. Patients will be evaluated at 13 clinical sites throughout North America and a genetics core will genotype all individuals for FTLD associated genes.

The study will be divided into 2 projects. The first project will be Preparing for Sporadic FTLD Clinical Trials and the second project will be a Longitudinal Assessment of Familial FTLD. Self-registration for an online registry will be available for patients and families with any FTLD syndrome. Eligible participants for research Projects 1 and 2 FTLD will be invited to a CRC site for clinical evaluations. All enrolled participants in both research projects will have a site visit consisting of a neurological exam, medical and family history, cognitive testing, and a blood draw.

Participants in Project 1 who have a diagnosis of Progressive Supranuclear Palsy Syndrome will have two additional assessments. A lumbar puncture (LP) will be performed for CSF collection, and an MRI scan of the brain will be done.

Participants in Project 2: Longitudinal Assessment of familial FTLD will return for a follow-up visit in 12 months; procedures at the follow-up visit will be identical to those at baseline. Additionally, asymptomatic participants will undergo MRI scans at both visits.

Clinical Study Identifier: NCT02365922

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Erik Roberson, MD

University of Alabama
Birmingham, AL United States
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Yvette Bordelon, MD, PhD

University of California, Los Angeles
Los Angeles, CA United States
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Irene Litvan, MD

University of California, San Diego
San Diego, CA United States
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Adam Boxer, MD, PhD

University of California, San Francisco
San Francisco, CA United States
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Neill Graff-Radford, M.D.

Mayo Clinic - Jacksonville
Jacksonville, FL United States
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Sandra Weintraub, PhD

Northwestern University
Chicago, IL United States
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Chiadi U Onyike, MD, MHS

Johns Hopkins University
Baltimore, MD United States
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Bradford C Dickerson, MD

Harvard University Massachusetts General Hospital
Charlestown, MA United States
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Davis S Knopman, M.D.

Mayo Clinic - Rochester
Rochester, MN United States
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Nupur Ghoshal, MD, PhD

Washington University
Saint Louis, MO United States
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Edward D Huey, MD

Columbia University
New York, NY United States
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Daniel Kaufer, MD

University of North Carolina
Chapel Hill, NC United States
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Brian Appleby, MD

Case Western Reserve University Hospitals Cleveland Medical Center
Cleveland, OH United States
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Murray Grossman, M.D.

University of Pennsylvania
Philadelphia, PA United States
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Diana Kerwin, MD

University of Texas Southwestern Medical Center
Dallas, TX United States
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Kimiko Domoto-Reilly, MD

University of Washington Harborview Medical Center
Seattle, WA United States
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Ian Mackenzie, MD

University of British Columbia
Vancouver, BC Canada
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Maria Carmela Tartaglia, MD, FRCPC

University of Toronto
Toronto, ON Canada
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