Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) — ARTFL  

Amyotrophic Lateral Sclerosis (ALS) Clinical Trial

Official title: Rare Diseases Clinical Research Network Advancing Research and Treatment for Frontotemporal Lobar Degeneration [ARTFL]: Research Projects 1 & 2

Status Completed

Clinical Trial Summary

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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Amyotrophic Lateral Sclerosis (ALS)
• Aphasia
• Aphasia, Primary Progressive
• Behavioral Variant Frontotemporal Dementia (bvFTD)
• Corticobasal Degeneration (CBD)
• Corticobasal Syndrome (CBS)
• Dementia
• Frontotemporal Dementia
• Frontotemporal Dementia (FTD)
• Frontotemporal Lobar Degeneration
• FTD With Amyotrophic Lateral Sclerosis (FTD/ALS)
• FTLD
• Motor Neuron Disease
• Nonfluent Variant Primary Progressive Aphasia (nfvPPA)
• Oligosymptomatic PSP (oPSP)
• Pick Disease of the Brain
• PPA Syndrome
• Progressive Supranuclear Palsy (PSP)
• Sclerosis
• Semantic Variant Primary Progressive Aphasia (svPPA)
• Supranuclear Palsy, Progressive
• Syndrome

• NCT number: NCT02365922
• Study type: Observational
• Source: University of California, San Francisco
• Status: Completed
• Start date: September 2014
• Completion date: September 2020