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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by University of California, San Francisco
Sponsor:
Collaborators:
National Center for Advancing Translational Science (NCATS)
National Institute of Neurological Disorders and Stroke (NINDS)
The Bluefield Project
Tau Consortium
Information provided by (Responsible Party):
University of California, San Francisco
ClinicalTrials.gov Identifier:
NCT02365922
First received: February 11, 2015
Last updated: March 1, 2017
Last verified: March 2017
  Purpose
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.

Condition
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)

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Rare Diseases Clinical Research Network Advancing Research and Treatment for Frontotemporal Lobar Degeneration [ARTFL]: Research Projects 1 & 2

Resource links provided by NLM:


Further study details as provided by University of California, San Francisco:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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
Plasma, serum, cell lines and cerebrospinal fluid will be retained by study investigators and stored at NIH-funded repositories.

Estimated Enrollment: 1560
Study Start Date: September 2014
Estimated Study Completion Date: February 2021
Estimated Primary Completion Date: September 2020 (Final data collection date for primary outcome measure)
Groups/Cohorts
Patients with FTLD or family members
Participants with FTLD syndrome diagnoses and/or strong family histories of FTLD.

Detailed 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.

  Eligibility

Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Patients with a diagnosis of a frontotemporal lobar degeneration (FTLD) syndrome, including progressive supranuclear palsy (PSP), semantic variant primary progressive aphasia (svPPA), and frontotemporal dementia with amyotrophic lateral sclerosis (FTD-ALS). Family members of patients with FTLD syndromes.
Criteria
  1. Inclusion Criteria:Must meet one of the following research diagnostic criteria for a Frontotemporal lobar degeneration (FTLD) syndrome: behavioral variant frontotemporal dementia (bvFTD), primary progressive aphasia (PPA), semantic variant primary progressive aphasia (svPPA), nonfluent variant primary progressive aphasia (nfvPPA), frontotemporal dementia with amyotrophic lateral sclerosis (FTD/ALS), amyotrophic lateral sclerosis alone, corticobasal syndrome (CBS), progressive supranuclear palsy (PSP) or oligosymptomatic PSP (oPSP), or have a strong family history of FTLD syndromes.
  2. Between 18 and 85 (inclusive) years of age.
  3. Able to walk (with assistance) at the time of enrollment.
  4. Have a reliable study partner who can provide an independent evaluation of functioning.
  5. Speak English or Spanish
  6. Have Mini Mental State Exam (MMSE) scores between 15 - 30 (inclusive).

Exclusion Criteria:

  1. Known presence of a structural brain lesion (e.g. tumor,cortical infarct) that could reasonably explain symptoms in a symptomatic participant without a known f-FTLD causing mutation.
  2. Known presence of an Alzheimer's disease causing mutation in PSEN1, PSEN2 or APP; or neuropathological evidence for Alzheimer's disease as a cause of syndrome (from brain biopsy).
  3. A previous history of Korsakoff encephalopathy, severe alcohol dependence (within 5 years of onset of dementia), frequent alcohol or other substance intoxication, or other neurological disorder (such as multiple sclerosis)
  4. Evidence through history or laboratory testing of B12 deficiency (B12 < 95% of local laboratory's normal value), hypothyroidism (TSH >150% of normal), HIV positive,renal failure (creatinine > 2), liver failure (ALT or AST > two times normal), respiratory failure (requiring oxygen), extra-axial brain tumor (with visible compression of the brain parenchyma), large cerebral infarct that could account for clinical syndrome, large confluent white matter lesions (grades 3 or 4, [107] significant systemic medical illnesses such as deteriorating cardiovascular disease;
  5. Current medication likely to affect CNS functions in the opinion of the site PI: long acting benzodiazepines such as diazepam (short-acting benzodiazepines are OK), non-SSRI antidepressants (SSRIs or trazodone are OK), no lithium, typical neuroleptics as listed in the Manual of Procedures, narcotics (codeine is OK, but hold 24 hours before neuropsychological testing), anticonvulsants (outside of therapeutic ranges), antihistamines (if taking greater than three times per week; hold 24 hours before neuropsychological testing).
  6. In the site investigator's opinion, the participant cannot complete sufficient key study procedures, or equivalent assessment of impairment level.
  7. For groups where MRI scans are planned procedures, any contraindication for MRI scanning, such as pacemaker or other implanted metals.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02365922

Contacts
Contact: Reilly Dever, BA 415-476-0670 Reilly.Dever@ucsf.edu

Locations
United States, Alabama
University of Alabama Recruiting
Birmingham, Alabama, United States, 35294
Contact: Emily McKinley, MSPH    205-996-3659    emckinley@uabmc.edu   
Principal Investigator: Erik Roberson, MD         
United States, California
University of California, Los Angeles Recruiting
Los Angeles, California, United States, 90095
Contact: Elvira Jimenez    310-478-3711 ext 40584    elvira@ucla.edu   
Principal Investigator: Yvette Bordelon, MD, PhD         
University of California, San Diego Recruiting
San Diego, California, United States, 92037
Contact: Kimberly Thomas, BA    858-822-5751    kkt008@ucsd.edu   
Principal Investigator: Irene Litvan, MD         
University of California, San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Reilly Dever, BA    415-476-0670    Reilly.Dever@ucsf.edu   
Principal Investigator: Adam Boxer, MD, PhD         
United States, Florida
Mayo Clinic - Jacksonville Recruiting
Jacksonville, Florida, United States, 32224
Contact: Dana Haley    904-953-9680    Haley.Dana@mayo.edu   
Principal Investigator: Neill Graff-Radford, M.D.         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Laura Martindale    312-503-5103    laura.martindale@northwestern.edu   
Principal Investigator: Sandra Weintraub, PhD         
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21287
Contact: Ann Fishman    410-502-5816    ann.fishman@jhu.edu   
Principal Investigator: Chiadi U Onyike, MD, MHS         
United States, Massachusetts
Harvard University Massachusetts General Hospital Recruiting
Charlestown, Massachusetts, United States, 02129
Contact: Samantha Krivensky    617-726-6205    skrivensky@mgh.harvard.edu   
Principal Investigator: Bradford C Dickerson, MD         
United States, Minnesota
Mayo Clinic - Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Ruth Kraft    507-538-9487    Kraft.Ruth@mayo.edu   
Principal Investigator: Bradley F Boeve, M.D.         
Principal Investigator: Davis S Knopman, M.D.         
United States, Missouri
Washington University Recruiting
St. Louis, Missouri, United States, 63110
Contact: Lynne Jones    314-362-8420    jonesly@wustl.edu   
Principal Investigator: Nupur Ghoshal, MD, PhD         
United States, New York
Columbia University Recruiting
New York, New York, United States, 10032
Contact: Masood Manoochehehri    212-305-5710    mm2626@cumc.columbia.edu   
Principal Investigator: Edward D Huey, MD         
United States, North Carolina
University of North Carolina Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Jennifer Wynne Taylor    919-966-8612    jwynne@neurology.unc.edu   
Principal Investigator: Daniel Kaufer, MD         
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104-4283
Contact: Christine Ray, MSW    215-349-5873    rayc@mail.med.upenn.edu   
Principal Investigator: Murray Grossman, M.D.         
Canada, British Columbia
University of British Columbia Recruiting
Vancouver, British Columbia, Canada, V6T 2B5
Contact: Pheth Sengdy, BSc, CCRP    604-822-7989    Pheth.Sengdy@vch.ca   
Principal Investigator: Robin Hsiung, MD         
Principal Investigator: Ian Mackenzie, MD         
Canada, Ontario
University of Toronto Recruiting
Toronto, Ontario, Canada, M5T 2S8
Contact: Behnaz Ghazanfari, MD, CCRP    416-603-5800 ext 5910    Behnaz.Ghazanfari@uhnresearch.ca   
Principal Investigator: Maria Carmela Tartaglia, MD, FRCPC         
Sponsors and Collaborators
University of California, San Francisco
National Center for Advancing Translational Science (NCATS)
National Institute of Neurological Disorders and Stroke (NINDS)
The Bluefield Project
Tau Consortium
Investigators
Principal Investigator: Adam L Boxer, MD, PhD Study PI
  More Information

Publications:

Responsible Party: University of California, San Francisco
ClinicalTrials.gov Identifier: NCT02365922     History of Changes
Other Study ID Numbers: ARTFL8101
1U54NS092089-01 ( US NIH Grant/Contract Award Number )
Study First Received: February 11, 2015
Last Updated: March 1, 2017

Keywords provided by University of California, San Francisco:
FTLD, PSP, CBD, PPA, FTD, FTD-ALS, CBS, ALS, svPPA, nfvPPA, oPSP

Additional relevant MeSH terms:
Syndrome
Sclerosis
Dementia
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Aphasia
Supranuclear Palsy, Progressive
Frontotemporal Dementia
Aphasia, Primary Progressive
Pick Disease of the Brain
Frontotemporal Lobar Degeneration
Primary Progressive Nonfluent Aphasia
Disease
Pathologic Processes
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Neurodegenerative Diseases
Neuromuscular Diseases
Spinal Cord Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations

ClinicalTrials.gov processed this record on March 28, 2017