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

Study 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.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Diseases, Study Type, Study Status, Phase, Gender

Study Status:

Active, currently recruiting

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Adam L Boxer, MD, PhD

Clinicaltrials.gov ID (11 digit #):

NCT02365922

Neals Affiliated?

No

Coordinating Center Contact Information

University of California, San Francisco
Alyssa Caplan, BA / .(JavaScript must be enabled to view this email address) / 415-476-0670
.(JavaScript must be enabled to view this email address) San Francisco, California 94158 United States

Full Study 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 (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 Sponsor:

University of California, San Francisco National Center for Advancing Translational Science (NCATS) National Institute of Neurological Disorders and Stroke (NINDS) The Bluefield Project to cure frontotemporal dementia Tau Consortium

Participant Duration:

Estimated Enrollment:

1560

Estimated Study Start Date:

08/31/2014

Estimated Study Completion Date:

08/31/2020

Posting Last Modified Date:

11/02/2016

Date Study Added to alsconsortium.org:

08/18/2015
  • More Information

    Alternative Study Name: Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)

    https://clinicalt...CT02365922&rank=1

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    85

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    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)
    -Between 18 and 85 (inclusive) years of age.
    -Able to walk (with assistance) at the time of enrollment.
    -Have a reliable study partner who can provide an independent evaluation of functioning.
    -Speak English or Spanish
    -Have Mini Mental State Exam (MMSE) scores between 15 - 30 (inclusive).

    Exclusion Criteria:
    -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.
    -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).
    -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)
    -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;
    -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).
    -In the site investigator's opinion, the participant cannot complete sufficient key study procedures.
    -For groups where MRI scans are planned procedures, any contraindication for MRI scanning, such as pacemaker or other implanted metals.

  • Site Contact Information

    Columbia University
    Masood Manoochehehri / .(JavaScript must be enabled to view this email address) / 212-305-5710
    New York, New York 10032
    United States

    Harvard University Massachusetts General Hospital
    Katie Kelly / .(JavaScript must be enabled to view this email address) / 617-726-6205
    Charlestown, Massachusetts 02129
    United States

    Johns Hopkins University
    Ann Fishman / .(JavaScript must be enabled to view this email address) / 410-502-5816
    Baltimore, Maryland 21287
    United States

    Mayo Clinic - Jacksonville
    Dana Haley / .(JavaScript must be enabled to view this email address) / 904-953-9680
    Jacksonville, Florida 32224
    United States

    Mayo Clinic - Rochester
    Dana Swenson-Dravis / .(JavaScript must be enabled to view this email address) / 507-583-0043
    Rochester, Minnesota 55905
    United States

    Northwestern University
    Laura Martindale / .(JavaScript must be enabled to view this email address) / 312-503-5103
    Chicago, Illinois 60611
    United States

    University of California, Los Angeles
    Elvira Jimenez / .(JavaScript must be enabled to view this email address) / 310-478-3711 ext 40584
    Los Angeles, California 90095
    United States

    University of California, San Diego
    Rachel Sinit, BA, CCRC / .(JavaScript must be enabled to view this email address) / 858-822-5786
    San Diego, California 92037
    United States

    University of California, San Francisco
    Alyssa Caplan, BA / .(JavaScript must be enabled to view this email address) / 415-476-0670
    San Francisco, California 94158
    United States

    University of North Carolina
    Lydia Hatfield / .(JavaScript must be enabled to view this email address) / 919-966-5039
    Chapel Hill, North Carolina 27599
    United States

    University of Pennsylvania
    Christine Ray, MSW / .(JavaScript must be enabled to view this email address) / 215-349-5873
    Philadelphia, Pennsylvania 19104
    United States

    Washington University
    Trish Stevenson / .(JavaScript must be enabled to view this email address) / 314-362-3613
    St. Louis, Missouri 63110
    United States

    University of Toronto
    Behmaz Ghazanfari, MD, CCRP / .(JavaScript must be enabled to view this email address) / 416-603-5800 ext 5910
    Toronto, Ontario M5T 2S8
    Canada

    University of British Columbia
    Pheth Sengdy, BSc, CCRP / .(JavaScript must be enabled to view this email address) / 604-822-7989
    Vancouver, British Columbia V6T 2B5
    Canada