ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)

Study Purpose:

ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) represents the formalized integration of ARTFL (U54 NS092089; funded through 2019) and LEFFTDS (U01 AG045390; funded through 2019) as a single North American research consortium to study FTLD for 2019 and beyond.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer with a Family History of ALS

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

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

Bradley Boeve, MD Mayo Clinic
Adam Boxer, MD, PhD University of California, San Francisco
Howie Rosen, MD University of California, San Francisco

Clinicaltrials.gov ID (11 digit #):

NCT04363684

Neals Affiliated?

No

Coordinating Center Contact Information

Mayo Clinic
Leah K Forsberg, PhD / .(JavaScript must be enabled to view this email address) / 507-293-9577
Hilary Heuer, PhD / .(JavaScript must be enabled to view this email address) / 415-476-6743
United States

Full Study Summary:

The ARTFL LEFFTDS Longitudinal Frontotemporal Dementia (ALLFTD) study aims to evaluate sporadic (s-) and familial (f-) frontotemporal lobar degeneration (FTLD) patients and asymptomatic family members of f-FTLD patients, characterizing the cohorts longitudinally and informing clinical trial design. The study has two arms: a "longitudinal arm" involving a comprehensive assessment of clinical, functional, imaging, and biofluid data collection annually, and a "biofluid-focused arm" involving limited clinical data to accompany biospecimen collection. For more information: https://www.allftd.org/

Study Sponsor:

Mayo Clinic

Participant Duration:

Estimated Enrollment:

2100

Estimated Study Start Date:

03/01/2020

Estimated Study Completion Date:

07/31/2024

Posting Last Modified Date:

07/29/2020

Date Study Added to alsconsortium.org:

05/13/2020
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Study Population
    Participants will have a referring diagnosis of an FTLD clinical syndrome or will be a member of a family with a strong family history of an FTLD syndrome.

    Criteria
    Longitudinal Arm Inclusion Criteria
    Familial FTLD (f-FTLD) participants (either is acceptable):

    • members of families in whom at least one member has a known disease-associated mutation in one of the major genes that cause f-FTLD: MAPT, GRN, C9orf72 (or other rare genes)
    • an autosomal dominant family history of a FTLD syndrome (without a known gene) verified by medical record review or well-documented family history including family members with a medical history consistent with FTLD or a related disorder.

    Sporadic FTLD (s-FTLD) participants:

    Sporadic participants should be symptomatic with no known family history nor a genetic mutation indicating f-FTLD. All sporadic participants must have an FTLD syndrome as a referring diagnosis; those determined by ALLFTD clinicians to have non-FTLD diagnoses will be excluded from longitudinal visits, but their baseline visit will be included in comparative datasets. For inclusion in the longitudinal follow-up, participants should meet research criteria for one of the following FTLD syndromes:

    • Progressive Supranuclear Palsy (PSP)
    • Semantic variant Primary Progressive Aphasia (svPPA)
    • Nonfluent variant Primary Progressive Aphasia (nfvPPA)
    • Corticobasal Degeneration (CBD)/Corticobasal Syndrome (CBS)
    • Behavioral variant Frontotemporal dementia (bvFTD)
    • Frontotemporal Dementia with Amyotrophic Lateral Sclerosis (FTD/ALS)

    Biofluid-Focused Arm Inclusion Criteria

    Participants enrolled in the biofluid arm may be either f-FTLD or s-FTLD. All general inclusion criteria apply. Participants should meet research criteria (as specified above) for any FTLD syndrome or meet familial FTLD inclusion criteria. Because the biofluid arm participants do not undergo the same detailed clinical and functional assessments required for the longitudinal arm, participants may be included regardless of primary language, as long as an appropriately translated consent is available.

    Exclusion Criteria:

    • Known presence of a structural brain lesion (e.g. tumor, cortical infarct) that could reasonably explain symptoms in a symptomatic participant.
    • Known presence of an Alzheimer's disease causing mutation in PSEN1, PSEN2 or APP; or biomarker evidence for Alzheimer's disease as a cause of the clinical syndrome.
    • 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.
    • Evidence through history or laboratory testing of uncorrected B12 deficiency (B12 < 95% of local laboratory's normal value), unregulated hypothyroidism (TSH >150% of normal), HIV positive, renal failure (creatinine > 2), liver failure (ALT or AST > two times normal), respiratory failure that requires supplemental oxygen, large confluent white matter lesions, significant systemic medical illnesses such as deteriorating cardiovascular disease.
    • Current medication likely to affect CNS functions in the opinion of the site PI.
    • In the site investigator's opinion, the participant cannot complete sufficient key study procedures. The participant may be enrolled into the biofluid-focused arm if they can tolerate a blood draw and short clinical exam, but must be able to complete at least 75% of study procedures for enrollment into the longitudinal arm.

  • Site Contact Information

    University of Alabama Birmingham
    Samantha Brown / .(JavaScript must be enabled to view this email address) / Birmingham, Alabama 35233
    United States

    University of California, Los Angeles
    Diana Chavez / .(JavaScript must be enabled to view this email address) / Los Angeles, California 90095
    United States

    University of California, San Diego
    Aishwarya Niraula / .(JavaScript must be enabled to view this email address) / San Diego, California 92093
    United States

    University of California San Francisco
    Lynn Bajorek / .(JavaScript must be enabled to view this email address) / Elise Ong / .(JavaScript must be enabled to view this email address) / San Francisco, California 91358
    United States

    Mayo Clinic Florida
    Ann Worthen / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32224
    United States

    Northwestern University
    Emma Pollner / .(JavaScript must be enabled to view this email address) / Chicago, Illinois 60611
    United States

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

    Massachusetts General Hospital
    Erin Krahn / .(JavaScript must be enabled to view this email address) / Boston, Massachusetts 02114
    United States

    Mayo Clinic Rochester
    Kevin Nelson / .(JavaScript must be enabled to view this email address) / 507-284-9295
    Rochester, Minnesota 55905
    United States

    Washinton University in St. Louis
    Tina Nolte / .(JavaScript must be enabled to view this email address) / Saint Louis, Missouri 63110
    United States

    Cleveland Clinic Lou Ruvo Center for Brain Health
    Las Vegas, Nevada 89106
    United States

    Columbia Unversity
    Masood Manoochehri / .(JavaScript must be enabled to view this email address) / New York, New York 10032
    United States

    University of North Carolina, Chapel Hill
    Karen Nicely / .(JavaScript must be enabled to view this email address) / Jessica Ferrall / .(JavaScript must be enabled to view this email address) / Chapel Hill, North Carolina 27514
    United States

    Case Western Reserve Medical Center
    Fran Lissemore / .(JavaScript must be enabled to view this email address) / 216-464-6203
    Cleveland, Ohio 44106
    United States

    University of Pennsylvania
    Jessica Bove / .(JavaScript must be enabled to view this email address) / Philadelphia, Pennsylvania 19104
    United States

    Nantz National Alzheimer Center Houston
    Victoria Arbones / .(JavaScript must be enabled to view this email address) / Houston, Texas 77030
    United States

    University of Washington
    CHristina Caso / .(JavaScript must be enabled to view this email address) / Seattle, Washington 98195
    United States

    University of British Columbia
    Yasmin Banga / .(JavaScript must be enabled to view this email address) / 604-822-5009
    Rachel Freid / .(JavaScript must be enabled to view this email address) / 604-827-1050
    Vancouver, British Columbia
    Canada

    University of Toronto
    Cristina Salvo / .(JavaScript must be enabled to view this email address) / 416-507-6880
    Behnaz Ghazanfari / .(JavaScript must be enabled to view this email address) / Toronto, Ontario
    Canada