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Longitudinal Early-onset Alzheimer's Disease Study Protocol

Recruiting
Conditions
Early Onset Alzheimer Disease
Alzheimer Disease
Mild Cognitive Impairment
Interventions
Drug: Florbetaben
Drug: Fluorodeoxyglucose
Registration Number
NCT03507257
Lead Sponsor
Indiana University
Brief Summary

The Longitudinal Early-onset Alzheimer's Disease Study (LEADS) is a non-randomized, natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment. Clinical, cognitive, imaging, biomarker, and genetic characteristics will be assessed across three cohorts: (1) early onset Alzheimer's Disease (EOAD) participants, (2) early onset non-Alzheimer's Disease (EOnonAD) participants, and (3) cognitively normal (CN) control participants.

Detailed Description

The LEADS study is a non-randomized, natural history, non-treatment study. Enrolled participants must be 40 - 64 (inclusive) years of age, with MCI due to AD or probable AD dementia (cognitively impaired participants) or have no significant memory impairment (cognitively normal \[CN\] participants).

Approximately 850 participants with cognitive impairment (650 with early onset Alzheimer's Disease \[EOAD\] and 200 with early onset non-Alzheimer's Disease \[EOnonAD\]) and 100 CN participants will be enrolled at approximately 20 sites in the United States. At approximately 5 sites outside of the United States, approximately 400 cognitively impaired participants and 10 CN participants will be enrolled. Cognitively impaired participants will take part in the study for 48+ months; CN participants will take part in the study for 24+ months.

Participants will undergo longitudinal clinical and cognitive assessments, computerized cognitive tests, biomarker and genetic tests, PET (FDG, amyloid and tau) and MRI brain scans, and optional cerebrospinal fluid (CSF) collection. Participants will be invited to consider autopsy brain donation

The primary objectives of the LEADS study are to:

* collect longitudinal assessments and biomarker data in individuals with early onset cognitive impairment (EOAD / EOnonAD) and cognitively normal (CN) controls;

* to compare baseline and longitudinal cognitive and functional characteristics, between EOAD and CN, and EOAD and Late Onset Alzheimer's Disease (LOAD) from the Alzheimer's Disease Neuroimaging Initiative (ADNI); and

* to study the associations of longitudinal clinical and cognitive assessments with multimodal imaging and biofluid markers that capture different elements of the AD pathophysiological cascade

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
850
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Early Onset non-Alzheimer's Disease (EOnonAD)Flortaucipir* Diagnosis of NIA-AA criteria of MCI due to AD or probable AD dementia * Amyloid negative status (florbetaben PET scan with no evidence of elevated amyloid as determined by a central read) * CDR score ≤ 1.0 * flortaucipir (18F-AV-1451) PET scanning
Early Onset non-Alzheimer's Disease (EOnonAD)Florbetaben* Diagnosis of NIA-AA criteria of MCI due to AD or probable AD dementia * Amyloid negative status (florbetaben PET scan with no evidence of elevated amyloid as determined by a central read) * CDR score ≤ 1.0 * flortaucipir (18F-AV-1451) PET scanning
Cognitively Normal (CN) ControlsFluorodeoxyglucose* Meets criteria for cognitively normal, based on an absence of significant impairment in cognitive functions and activities of daily living * Mini-Mental State Exam score between 26-30 * CDR score = 0 * flortaucipir (18F-AV-1451) PET scanning
Early Onset Alzheimer's Disease (EOAD)Florbetaben* Diagnosis of NIA-AA criteria of MCI due to AD or probable AD dementia * Amyloid positive status (florbetaben PET scan with evidence of elevated amyloid as determined by a central read) * CDR score ≤ 1.0 * flortaucipir (18F-AV-1451) PET scanning
Cognitively Normal (CN) ControlsFlorbetaben* Meets criteria for cognitively normal, based on an absence of significant impairment in cognitive functions and activities of daily living * Mini-Mental State Exam score between 26-30 * CDR score = 0 * flortaucipir (18F-AV-1451) PET scanning
Early Onset non-Alzheimer's Disease (EOnonAD)Fluorodeoxyglucose* Diagnosis of NIA-AA criteria of MCI due to AD or probable AD dementia * Amyloid negative status (florbetaben PET scan with no evidence of elevated amyloid as determined by a central read) * CDR score ≤ 1.0 * flortaucipir (18F-AV-1451) PET scanning
Early Onset Alzheimer's Disease (EOAD)Flortaucipir* Diagnosis of NIA-AA criteria of MCI due to AD or probable AD dementia * Amyloid positive status (florbetaben PET scan with evidence of elevated amyloid as determined by a central read) * CDR score ≤ 1.0 * flortaucipir (18F-AV-1451) PET scanning
Cognitively Normal (CN) ControlsFlortaucipir* Meets criteria for cognitively normal, based on an absence of significant impairment in cognitive functions and activities of daily living * Mini-Mental State Exam score between 26-30 * CDR score = 0 * flortaucipir (18F-AV-1451) PET scanning
Primary Outcome Measures
NameTimeMethod
Rate of change in cognition as measured by the Alzheimer's Disease Assessment Scale-Cognitive (ADAS-Cog13)Month 0, Month 12, Month 24, Month 36 (EOAD/EOnonAD only) and Month 48 (EOAD/EOnonAD only)

The ADAS-Cog is an in-person examiner-administered, structured scale that evaluates memory (word recall, word recognition), reasoning (following commands), language (naming, comprehension), orientation, ideational praxis (placing letter in envelope) and constructional praxis (copying geometric designs). Ratings of spoken language, language comprehension, word finding difficulty, and ability to remember test instructions are also obtained.

Secondary Outcome Measures
NameTimeMethod
Neurodegeneration as measured by fluorodeoxyglucose (FDG) Positron Emission Tomography (PET) imaging compared to magnetic resonance imaging (MRI)Month 12 (EOnonAD only) and Month 24 (CN only)
Rate of change in cognition as measured by the Clinical Dementia Rating - Sum of Boxes (CDR-SB)CN participants: Month 0 and Month 24; EOAD/EOnonAD participants: Month 0, Month 12, Month 24, Month 36 and Month 48

The CDR is a semi-structured interview of the informant and participant that assesses for impairment in 8 areas of functioning - memory, orientation, judgment and problem solving, community affairs, home and hobbies, personal care, behavior, personality, and language.

Change in tau deposition as measured by flortaucipir (18F-AV-1451) Positron Emission Tomography (PET) imagingMonth 0, Month 12 (EOAD only), Month 24 (EOAD only) and Month 36 (EOAD and EOnonAD amyloid positive participants only)
Change in amyloid deposition as measured by florbetaben using Positron Emission Tomography (PET) imagingMonth 0, Month 12 (EOAD only), Month 24 (EOAD only) and Month 36 (EOAD/EOnonAD only
Change in plasma biomarkersCN participants: Month 0, Month 12 and Month 24; EOAD/EOnonAD participants: Month 0, Month 12, Month 24, Month 36 and Month 48
Change in brain structure using magnetic resonance imaging (MRI)CN participants: Month 0 and Month 24; EOAD/EOnonAD participants: Month 0, Month 12, Month 24 and Month 36
Change in cerebrospinal fluid (CSF) biomarkersCN participants: Month 0 and Month 24; EOAD/EOnonAD participants: Month 0, Month 12, Month 24, Month 36

Trial Locations

Locations (23)

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

Stanford University

🇺🇸

Palo Alto, California, United States

University of California, San Francisco

🇺🇸

San Francisco, California, United States

Georgetown University

🇺🇸

Washington, District of Columbia, United States

Mayo Clinic, Jacksonville

🇺🇸

Jacksonville, Florida, United States

Wien Center

🇺🇸

Miami Beach, Florida, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

Indiana University

🇺🇸

Indianapolis, Indiana, United States

Johns Hopkins University

🇺🇸

Baltimore, Maryland, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Mayo Clinic, Rochester

🇺🇸

Rochester, Minnesota, United States

Washington University, St. Louis

🇺🇸

Saint Louis, Missouri, United States

Columbia University

🇺🇸

New York, New York, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Butler Hospital

🇺🇸

Providence, Rhode Island, United States

Houston Methodist Hospital

🇺🇸

Houston, Texas, United States

Fleni

🇦🇷

Buenos Aires, Argentina

Amsterdam UMC

🇳🇱

Amsterdam, Netherlands

Hospital de la Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

Lund University

🇸🇪

Malmö, Sweden

University College London

🇬🇧

London, United Kingdom

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