ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)
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|ClinicalTrials.gov Identifier: NCT04363684|
Recruitment Status : Recruiting
First Posted : April 27, 2020
Last Update Posted : May 12, 2023
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|Condition or disease|
|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|
|Study Type :||Observational|
|Estimated Enrollment :||2100 participants|
|Official Title:||ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)|
|Actual Study Start Date :||March 1, 2020|
|Estimated Primary Completion Date :||July 2024|
|Estimated Study Completion Date :||July 2024|
Annual clinic visits throughout the length of the study.
Single clinic visit.
- Change in Brain Volumes [ Time Frame: Baseline, 1 Year, 2 Year, 3 Year, 4 Year, 5 Year ]Compare rates of change in whole brain and regional volumes between asymptomatic f-FTLD and symptomatic f- and s-FTLD, measured using MRI.
- Change in NIH Examiner Executive Composite Score [ Time Frame: Baseline, 1 Year, 2 Year, 3 Year, 4 Year, 5 Year ]Evaluate change in NIH Examiner Executive Composite Score in asymptomatic f-FTLD.
- Change in Multidomain Impairment Rating (MIR) Scale [ Time Frame: Baseline, 1 Year, 2 Year, 3 Year, 4 Year, 5 Year ]Annual change in MIR score (total score 0-3), which is a new global scale for FTLD that incorporates behavioral, cognitive, and motor dysfunction in the rating.
- Plasma Neurofilament Light Chain Analysis [ Time Frame: 5 years ]Annual blood samples will be collected to detect changes in plasma neurofilament light chain concentrations
Biospecimen Retention: Samples With DNA
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||Yes|
|Sampling Method:||Non-Probability Sample|
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.
- 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.
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): NCT04363684
|Contact: Leah K Forsberg, PhDfirstname.lastname@example.org|
|Contact: Hilary Heuer, PhDemail@example.com|
|Principal Investigator:||Bradley Boeve, MD||Mayo Clinic|
|Principal Investigator:||Adam Boxer, MD, PhD||University of California, San Francisco|
|Principal Investigator:||Howie Rosen, MD||University of California, San Francisco|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Bradley Boeve, Principal Investigator, Mayo Clinic|
|Other Study ID Numbers:||
U19AG063911 ( U.S. NIH Grant/Contract )
|First Posted:||April 27, 2020 Key Record Dates|
|Last Update Posted:||May 12, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||De-identified subject level data will be shared upon approved data request.|
|Time Frame:||De-identified data will be available for at least the duration of the study.|
Interested researchers must complete a data request through the ALLFTD website. All data requests will be reviewed by a committee for evaluation of scientific merit and feasibility. Please consult the website for additional information regarding this process (https://www.allftd.org/policies).
Approved requests will be delivered in a de-identified manner.
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Aphasia, Primary Progressive
Pick Disease of the Brain
Supranuclear Palsy, Progressive
Frontotemporal Lobar Degeneration
Central Nervous System Diseases
Nervous System Diseases
Spinal Cord Diseases