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


Brief 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.


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

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

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

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.


  • University of California, Los Angeles, Los Angeles, California, United States, 90095
  • University of California, San Diego, San Diego, California, United States, 92037
  • University of California, San Francisco, San Francisco, California, United States, 94158
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