Summary

Eligibility
for people ages 18 years and up (full criteria)
Healthy Volunteers
healthy people welcome
Location
at San Francisco, California and other locations
Dates
study started
completion around
Principal Investigator
by Adam Boxer, MD, PhDHowie Rosen, MD
Headshot of Adam Boxer
Adam Boxer
Headshot of Howie Rosen
Howie Rosen

Description

Summary

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.

Details

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/

Keywords

Frontotemporal Lobar Degeneration (FTLD), Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), 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, Oligosymptomatic PSP (oPSP), C9orf72, GRN Related Frontotemporal Dementia, MAPT Gene Mutation, TBK1 Gene Mutation, Oligosymptomatic Progressive Supranuclear Palsy, Dementia, Motor Neuron Disease, Aphasia, Frontotemporal Dementia, Primary Progressive Aphasia, Pick Disease of the Brain, Progressive Supranuclear Palsy, Frontotemporal Lobar Degeneration, Corticobasal Degeneration, Sclerosis

Eligibility

For people ages 18 years and up

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:

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.

Locations

  • UCSF accepting new patients
    San Francisco California 91358 United States
  • University of California, Los Angeles accepting new patients
    Los Angeles California 90095 United States

Lead Scientists at UCSF

  • Adam Boxer, MD, PhD
    Adam L. Boxer, MD, PhD is the Endowed Professor in Memory and Aging in the Department of Neurology, Weill Institute of Neuroscience, University of California, San Francisco (UCSF). He received his MD and PhD from the New York University Medical Center Medical Scientist Training Program, completed a Neurology Residency at Stanford and a Neurobehavior Fellowship at UCSF.
  • Howie Rosen, MD
    Professor, Neurology, School of Medicine. Authored (or co-authored) 364 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Mayo Clinic
Links
ALLFTD Study Website
ID
NCT04363684
Study Type
Observational
Participants
Expecting 2100 study participants
Last Updated