Clinical trial NCT03088956: Cognitive, Behavioral, and Functional Change in Behavioral Variant Frontotemporal Dementia (bvFTD)
Official title: A Study to Model Rates of Change on Neuropsychological Test Measures in Subjects Diagnosed With Behavioral Variant Frontotemporal Dementia and Healthy Subjects
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Frontotemporal Dementia
Behavioral Variant Frontotemporal Dementia
Keywords: Longitudinal
Caregiver
Cognitive assessments
Behavioral assessments
Functional assessments
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2017-03-24
Enrollment: 47
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Academic centers, Hospitals, Clinics

Brief summary: The objectives of the study are to; (1) estimate the change in disease -related cognitive decline over 1 year on a battery of cognitive tests administered to participants with early-stage symptomatic Behavioral Variant Frontotemporal Dementia (bvFTD) phenotypic variant; (2) identify the cognitive test or brief battery of cognitive tests which are the most sensitive to detect bvFTD progression; (3) determine the optimal schedule of administration of cognitive tests to detect bvFTD progression; (4) evaluate the relationship between cognitive tests and measures of behavior, function, caregiver's burden, quality of life (QOL); and (5) obtain blood samples for genetic and exploratory biomarkers correlations.


Eligibility criteria: Key Inclusion Criteria for Participants with behavioral variant frontotemporal dementia (bvFTD):

* Must speak and understand oral and written English.
* Must have probable bvFTD.
* Must have a global Clinical Dementia Rating (CDR) score of 0.5-1 and a Mini Mental State Examination (MMSE) score ≥20 to reflect early stages of disease.
* Must have 1 informant/caregiver who, in the Investigator's judgment, has frequent and sufficient contact with the participant as to be able to provide accurate information about the participant's behaviors as well as cognitive and functional abilities.
* Must be ambulatory or able to walk with assistance and not be institutionalized.

Key Inclusion Criteria for Healthy Participants:

* Must speak and understand oral and written English.
* Must be in good general health determined by Investigator.

Key Exclusion Criteria for Participants with bvFTD:

* Concomitant motor neuron disease with limb or bulbar weakness which, in the opinion of the Investigator, may affect performance over the course of the study. Participants with bvFTD with motor neuron disease are otherwise allowed to participate.
* Known presence of a structural brain lesion that could reasonably explain symptoms.
* Diagnosis of Alzheimer's disease and/or 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.
* History of other acute or chronic neurological or psychiatric conditions that are unrelated and may confound a diagnosis of bvFTD and that, in the opinion of the Investigator, may affect cognition, behavior, or ability to complete the study.
* History of severe alcohol or substance abuse.
* History of disorders that could confound a diagnosis of bvFTD.
* Use of allowed chronic medications at doses that have not been stable for at least 4 weeks prior to Screening.
* Current use of psychoactive medication that will interfere with accurate assessment of cognition (as assessed by the Investigator)

Key Exclusion Criteria for Healthy Participants:

* History of alcohol or substance abuse.
* Current use of psychoactive medication that will interfere with accurate assessment of cognition (as assessed by the Investigator).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Annualized rate of change in International Shopping List Test (ISLT) - Immediate Recall
timeFrame: Up to 12 Months
measure: Annualized rate of change in Detection Test
timeFrame: Up to 12 Months
measure: Annualized rate of change in Identification Test
timeFrame: Up to 12 Months
measure: Annualized rate of change in One Back Test
timeFrame: Up to 12 Months
measure: Annualized rate of change in Groton Maze Learning Test
timeFrame: Up to 12 Months
measure: Annualized rate of change in ISLT - Delayed Recall
timeFrame: Up to 12 Months
measure: Annualized rate of change in Social Emotional Cognition Test (SECT)
timeFrame: Up to 12 Months
measure: Annualized rate of change in Letter Fluency
timeFrame: Up to 12 Months
measure: Annualized rate of change in Category Fluency
timeFrame: Up to 12 Months
measure: Annualized rate of change in Digit Symbol Coding (DSC)
timeFrame: Up to 12 Months
measure: Annualized rate of change in Letter Number Sequencing (LNS)
timeFrame: Up to 12 Months
measure: Annualized rate of change in Mini Mental State Examination (MMSE)
timeFrame: Up to 12 Months
measure: Change from Day 1 in International Shopping List Test (ISLT) - Immediate Recall
timeFrame: Up to 12 Months
measure: Change from Day 1 in Detection Test
timeFrame: Up to 12 Months
measure: Change from Day 1 in Identification Test
timeFrame: Up to 12 Months
measure: Change from Day 1 in One Back Test
timeFrame: Up to 12 Months
measure: Change from Day 1 in Groton Maze Learning Test
timeFrame: Up to 12 Months
measure: Change from Day 1 in ISLT- Delayed Recall
timeFrame: Up to 12 Months
measure: Change from Day 1 in SECT
timeFrame: Up to 12 Months
measure: Change from Day 1 in Letter Fluency
timeFrame: Up to 12 Months
measure: Change from Day 1 in Category Fluency
timeFrame: Up to 12 Months
measure: Change from Day 1 in DSC
timeFrame: Up to 12 Months
measure: Change from Day 1 in LNS
timeFrame: Up to 12 Months
measure: Change from Day 1 in MMSE
timeFrame: Up to 12 months
measure: Actual values from Day 1 in Neurophsychic Inventory (NPI)
timeFrame: Up to 12 Months
measure: Change from Day 1 in NPI
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Neurophsychic Inventory Questionnaire (NPI-Q)
timeFrame: Up to 12 Months
measure: Change from Day 1 in NPI-Q
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Revised Self-Monitoring Scale
timeFrame: Up to 12 Months
measure: Change from Day 1 in Revised Self-Monitoring Scale
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Apathy Inventory
timeFrame: Up to 12 Months
measure: Change from Day 1 in Apathy Inventory
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Family Accommodation Scale for Obsessive Compulsive Disorder - Self Rated (FAS-SR)
timeFrame: Up to 12 Months
measure: Change from Day 1 in FAS-SR
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Behavior Rating Scale
timeFrame: Up to 12 Months
measure: Change from Day 1 in Behavior Rating Scale
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Functional Activities Questionnaire (FAQ)
timeFrame: Up to 12 Months
measure: Change from Day 1 in FAQ
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Clinical Global Impression of Change (CGIC)
timeFrame: Up to 12 Months
measure: Change from Day 1 in CGIC
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Clinical Dementia Rating-Frontotemporal Lobar Degeneration (CDR-FTLD)
timeFrame: Up to 12 Months
measure: Change from Day 1 in CDR-FTLD
timeFrame: Up to 12 Months
measure: Actual values from Day 1 in Unified Parkinson's Disease Rating Scale (UPDRS) Part III
timeFrame: Up to 12 Months
measure: Change from Day 1 in UPDRS Part III
timeFrame: Up to 12 Months
measure: Actual values from Day 1 of caregiver's Quality of Life (QOL) in Zarit Caregiver Burden Interview
timeFrame: Up to 12 Months
measure: Change from Day 1 of caregiver's QOL in Zarit Caregiver Burden Interview
timeFrame: Up to 12 Months
measure: Actual values from Day 1 of QOL in Geriatric Depression Scale- Short Form (GDS-SF)
timeFrame: Up to 12 Months
measure: Change from Day 1 of QOL in GDS-SF
timeFrame: Up to 12 Months
measure: Actual values from Day 1 of caregiver's QOL in Caregiver Assessment Measure
timeFrame: Up to 12 Months
measure: Change from Day 1 of caregiver's QOL in Caregiver Assessment Measure
timeFrame: Up to 12 Months
measure: Actual values from Day 1 of QOL in Quality of Life in Neurological Disorders (NeuroQOL)
timeFrame: Up to 12 Months
measure: Change from Day 1 of QOL in Quality of Life in NeuroQOL
timeFrame: Up to 12 Months
measure: Actual values from Day 1 of QOL in EuroOol 5 Dimensions Questionnaire (EQ-5D)
timeFrame: Up to 12 Months
measure: Change from Day 1 of QOL in EQ-5D
timeFrame: Up to 12 Months
measure: Actual values from Day 1 of QOL in Affect and Activity Indicators of QOL (AAIQOL)
timeFrame: Up to 12 Months
measure: Change from Day 1 of QOL in AAIQOL
timeFrame: Up to 12 Months
measure: Actual values from Day 1 for exploratory biomarkers
timeFrame: Baseline, Month 12
measure: Change from Day 1 for exploratory blood biomarkers
timeFrame: Baseline, Month 12
measure: bvFTD genetic mutations identification in whole blood samples
timeFrame: Baseline, Month 12
measure: Potential identification of genetic biomarkers associated with bvFTD from DNA/RNA in whole blood samples
timeFrame: Baseline, Month 12
measure: Actual values at Day 1 in Clinical Global Impression of Severity (CGIS)
timeFrame: Day 1
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Research Site
state: Alabama
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Boca Raton
country: United States
facility: Research Site
state: Florida
city: Boca Raton
country: United States
facility: Research Site
state: Florida
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Elk Grove Village
country: United States
facility: Research Site
state: Illinois
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Rochester
country: United States
facility: Research Site
state: Minnesota
city: Columbus
country: United States
facility: Research Site
state: Ohio
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Knoxville
country: United States
facility: Research Site
state: Tennessee
city: Seattle
country: United States
facility: Research Site
state: Washington
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: