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Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)

2015-05-28 22:37:25 | BioPortfolio

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.

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.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Conditions

FTLD

Location

University of California, Los Angeles
Los Angeles
California
United States
90095

Status

Recruiting

Source

University of California, San Francisco

Results (where available)

View Results

Links

Published on BioPortfolio: 2015-05-28T22:37:25-0400

Clinical Trials [7 Associated Clinical Trials listed on BioPortfolio]

Serotonergic Function and Behavioural and Psychological Symptoms of Frontotemporal Dementia

Frontotemporal lobar degeneration(FTLD) is a common cause of early-onset dementia. FTLD is characterized multiple behavioral symptoms including mental rigidity, irritability, emotional bl...

Multidisciplinary and Personalized Care of Behavioral Disorders in Frontotemporal Lobar Degeneration.

Fronto-Temporal Lobar Degeneration (FTLD) is neurodegenerative disease which comes in three clinical categories: Fronto-Temporal Dementia behavioral variant (FTD), Primary Progressive Apha...

A 52 Week Open Label Trial of Memantine for Frontotemporal Lobar Degeneration

This is a 52-week, multicenter, open label trial of memantine (Namenda) for frontotemporal lobar degeneration (FTLD). The goal is to determine the safety and tolerability of this FDA-appr...

Diagnosing Frontotemporal Lobar Degeneration

To establish diagnostic tools to make an accurate clinical and pathological diagnosis of patients with clinical FTLD syndromes

Natural History Characterization in Symptomatic and Asymptomatic Progranuline Gene Mutation Carriers

The purpose of this study is to investigate whether cognitive deficits, structural and functional changes can be detected before symptom onset in presymptomatic progranuline mutation carri...

PubMed Articles [17 Associated PubMed Articles listed on BioPortfolio]

Serum neurofilament light chain is a discriminative biomarker between frontotemporal lobar degeneration and primary psychiatric disorders.

Due to the significant clinical overlap between frontotemporal lobar degeneration (FTLD) spectrum disorders and late-onset primary psychiatric disorders (PPD), diagnostic biomarkers reflecting the dif...

Suppression of Progranulin Expression Leads to Formation of Intranuclear TDP-43 Inclusions In Vitro: A Cell Model of Frontotemporal Lobar Degeneration.

Mutations in the GRN gene coding for progranulin (PGRN) are responsible for many cases of familial frontotemporal lobar degeneration (FTLD) with TAR DNA-binding protein 43 (TDP-43)-positive inclusions...

Behavioural impairments in mice of a novel FUS transgenic line recapitulate features of frontotemporal lobar degeneration.

Multiple clinical and experimental evidence suggest that ALS and FTLD are members of a disease continuum. Pathological FUS inclusions have been observed in subsets of patients with these diseases but ...

Cancer-related genes and ALS.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that is characterized by the progressive degeneration of both upper motor neurons in the motor cortex and lower motor neurons in the...

Progranulin deficiency leads to reduced glucocerebrosidase activity.

Mutation in the GRN gene, encoding the progranulin (PGRN) protein, shows a dose-dependent disease correlation, wherein haploinsufficiency results in frontotemporal lobar degeneration (FTLD) and comple...

Medical and Biotech [MESH] Definitions

Heterogeneous group of neurodegenerative disorders characterized by frontal and temporal lobe atrophy associated with neuronal loss, gliosis, and dementia. Patients exhibit progressive changes in social, behavioral, and/or language function. Multiple subtypes or forms are recognized based on presence or absence of TAU PROTEIN inclusions. FTLD includes three clinical syndromes: FRONTOTEMPORAL DEMENTIA, semantic dementia, and PRIMARY PROGRESSIVE NONFLUENT APHASIA.

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