Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) (ARTFL)
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| ClinicalTrials.gov Identifier: NCT02365922 |
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Recruitment Status :
Completed
First Posted : February 19, 2015
Last Update Posted : May 21, 2021
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| Condition or disease |
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| 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) |
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 Type : | Observational |
| Actual Enrollment : | 1489 participants |
| 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 |
| Actual Study Start Date : | September 2014 |
| Actual Primary Completion Date : | August 2020 |
| Actual Study Completion Date : | September 2020 |
| Group/Cohort |
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Patients with FTLD or family members
Participants with FTLD syndrome diagnoses and/or strong family histories of FTLD.
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- 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.
- 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
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| Ages Eligible for Study: | 18 Years to 85 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
| Sampling Method: | Non-Probability Sample |
- 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.
- 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, or equivalent assessment of impairment level.
- For groups where MRI scans are planned procedures, any contraindication for MRI scanning, such as pacemaker or other implanted metals.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02365922
Show 17 study locations
| Principal Investigator: | Adam L Boxer, MD, PhD | Study PI |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | University of California, San Francisco |
| ClinicalTrials.gov Identifier: | NCT02365922 |
| Other Study ID Numbers: |
ARTFL8101 1U54NS092089-01 ( U.S. NIH Grant/Contract ) |
| First Posted: | February 19, 2015 Key Record Dates |
| Last Update Posted: | May 21, 2021 |
| Last Verified: | May 2021 |
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FTLD, PSP, CBD, PPA, FTD, FTD-ALS, CBS, ALS, svPPA, nfvPPA, oPSP |
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Dementia Frontotemporal Dementia Pick Disease of the Brain Frontotemporal Lobar Degeneration Motor Neuron Disease Amyotrophic Lateral Sclerosis Aphasia Aphasia, Primary Progressive Supranuclear Palsy, Progressive Corticobasal Degeneration Syndrome Sclerosis Disease Pathologic Processes Brain Diseases |
Central Nervous System Diseases Nervous System Diseases Neurocognitive Disorders Mental Disorders Tauopathies Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Speech Disorders Language Disorders Communication Disorders Neurobehavioral Manifestations |