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.
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.
Study Type
OBSERVATIONAL
Enrollment
1,489
University of Alabama
Birmingham, Alabama, United States
University of California, Los Angeles
Los Angeles, California, United States
University of California, San Diego
San Diego, California, United States
University of California, San Francisco
San Francisco, California, United States
Mayo Clinic - Jacksonville
Jacksonville, Florida, United States
Northwestern University
Chicago, Illinois, United States
Johns Hopkins University
Baltimore, Maryland, United States
Harvard University Massachusetts General Hospital
Charlestown, Massachusetts, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Washington University
St Louis, Missouri, United States
...and 7 more locations
Scores of UDS FTLD Module Tests
Neuropsychological test scores from the Uniform Data Set FTLD Module will be collected and compared across patient populations.
Time frame: Baseline, 12 mo.
Progressive Supranuclear Palsy Rating Scale (PSPRS)
Scores will be compared among patient populations
Time frame: Baseline
Neuroimaging
In asymptomatic family members of FTLD patients, changes from baseline neuroimaging will be assessed 12 months later.
Time frame: Baseline; 12 months
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