Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) (ARTFL)
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|ClinicalTrials.gov Identifier: NCT02365922|
Recruitment Status : Completed
First Posted : February 19, 2015
Last Update Posted : May 21, 2021
|Condition or disease|
|FTLD Progressive Supranuclear Palsy (PSP) Frontotemporal Dementia (FTD) Corticobasal Degeneration (CBD) PPA Syndrome Behavioral Variant Frontotemporal Dementia (bvFTD) Semantic Variant Primary Progressive Aphasia (svPPA) Nonfluent Variant Primary Progressive Aphasia (nfvPPA) FTD With Amyotrophic Lateral Sclerosis (FTD/ALS) Amyotrophic Lateral Sclerosis (ALS) Oligosymptomatic PSP (oPSP) Corticobasal Syndrome (CBS)|
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|
|Actual Enrollment :||1489 participants|
|Official Title:||Rare Diseases Clinical Research Network Advancing Research and Treatment for Frontotemporal Lobar Degeneration [ARTFL]: Research Projects 1 & 2|
|Actual Study Start Date :||September 2014|
|Actual Primary Completion Date :||August 2020|
|Actual Study Completion Date :||September 2020|
Patients with FTLD or family members
Participants with FTLD syndrome diagnoses and/or strong family histories of FTLD.
- Scores of UDS FTLD Module Tests [ Time Frame: Baseline, 12 mo. ]Neuropsychological test scores from the Uniform Data Set FTLD Module will be collected and compared across patient populations.
- Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: Baseline ]Scores will be compared among patient populations
- Neuroimaging [ Time Frame: Baseline; 12 months ]In asymptomatic family members of FTLD patients, changes from baseline neuroimaging will be assessed 12 months later.
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02365922
|Principal Investigator:||Adam L Boxer, MD, PhD||Study PI|