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Longitudinal Early-onset Alzheimer's Disease Study Protocol

NCT03507257 · Indiana University
In plain English

Click the button to translate this study into plain language — what it is, who qualifies, and what participation looks like.

About this study
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
Eligibility criteria
Inclusion Criteria for Cognitively Impaired (EOAD and EOnonAD) Cohorts Only: 1. Meets NIA-AA criteria for MCI due to AD or probable AD dementia 2. Have a global CDR score ≤ 1.0 3. Have capacity to provide informed consent (IC) or has a legal authorized representative or guardian who provides IC 4. Age between 40-64 years (inclusive) at the time of consent 5. Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant (e.g., family member, significant other, friend, caregiver) who is generally aware of the participants' daily activities and can provide information about the participant's cognitive and functional performance. If the participant does not have a study partner who spends at least 10 face-to-face hours per week, other arrangements for identifying a viable study partner will be granted on a case-by-case basis by the Site PI 6. Willing and able to complete longitudinal study procedures aside from LP which is an optional procedure 7. Not pregnant or lactating. Women must be two years post-menopausal, be surgically sterile, or have a negative pregnancy test prior to each PET scan 8. Fluent in English or Spanish if enrolled in the U.S. 9. Fluent in English, Spanish, Dutch or Swedish for sites outside the U.S., according to site's spoken language(s). Inclusion Criteria for Cognitively Normal (CN) Cohort Only: 1. Meets criteria for cognitively normal, based on an absence of significant impairment in cognitive functions or activities of daily living 2. Have a global CDR score = 0 3. Have capacity to provide informed consent 4. Have a Mini-Mental State Exam score between 26-30 (inclusive). Exceptions may be made for participant with less than 8 years of education at the discretion of the Site PI 5. Age between 40-64 years (inclusive) at the time of consent 6. Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant (e.g., family member, significant other, friend, caregiver) who is generally aware of the participants' daily activities and can provide information about the participant's cognitive and functional performance. If the participant does not have a study partner who spends 10 face-to-face hours per week, other arrangements for identifying a viable study partner will be granted on a case-by-case basis by the Site PI 7. Willing and able to complete longitudinal study procedures aside from LP which is an optional procedure 8. Not pregnant or lactating. Women must be two years post-menopausal, be surgically sterile, or have a negative pregnancy test prior to each PET scan 9. Fluent in English or Spanish if enrolled in the U.S. 10. Fluent in English, Spanish, Dutch or Swedish for sites outside the U.S., according to site's spoken language(s). Exclusion Criteria for all (EOAD, EOnonAD and CN) cohorts: 1. Meets core clinical criteria for non-AD dementia 2. Two or more first degree relatives with a history of early-onset dementia suggestive of autosomal dominant transmission, unless known pathogenic mutations in APP, PSEN1, PSEN2, MAPT, GRN and C9ORF72 have been excluded 3. Known CLIA certified mutation in an ADAD gene (APP, PSEN1, PSEN2), or other autosomal dominant genes associated with other neurodegenerative disorders (MAPT, GRN, C9ORF72) 4. Contraindications to 3T MRI (e.g., claustrophobia, pacemaker, select aneurismal clip, artificial heart valve, select ear implants, select stents incompatible with 3T MRI, metal fragments or foreign objects in the eyes, skin or body, etc.) 5. Lifetime medical history of a brain disorder other than the disorder causing dementia except for headache (exceptions are allowed at the discretion of the Site PI - e.g., seizure disorder thought to be due to EOAD). 6. MRI scan with evidence of infection or focal lesions, cortical strokes, multiple lacunes (single lacune is allowable unless it meets criteria for strategic lacune affecting cognition) 7. Any significant systemic illness or unstable medical condition, which could lead to difficulty complying with the protocol (at the discretion of the Site PI) 8. Research radiation exposure will be assessed by the study physician. If the candidate participant has had more than one nuclear medicine study in the prior 12 months for research-related purposes, study inclusion will require approval from the PET Core 9. Investigational agents are prohibited 30 days prior to entry 10. Previous enrollment in a therapeutic trial targeting amyloid or tau. 11. Participation in other clinical studies with neuropsychological measures, with the exception of participants who are co-enrolled in the NACC Uniform Data Set (UDS) protocol (Note: This criterion is intended to reduce repeat measures effects during neuropsychological testing. Exceptions are allowed at the discretion of the Site PI) 12. Lifetime history of schizophrenia spectrum disorders (DSM-5 criteria) 13. Current history (in previous 12 months) of DSM-5 diagnosis of mania, bipolar disorder with or without psychotic features 14. Current history (in previous 6 months) of moderate or severe substance abuse (nicotine or caffeine is allowed) 15. Suicidal behaviors in the past 12 months or active suicidal ideations 16. Residing in a 24-hour care skilled nursing facility (at the time of screening) 17. (For optional lumbar puncture procedure only): a. Clinical laboratory values must be within normal limits or, if abnormal, must be judged to be not clinically significant by the Site PI i. Platelet count \<100,000/ml ii. INR\>1.2 iii. Abnormal PT or PTT at screening b. Contraindications to the procedure, including but not limited to severe degenerative joint disease, deformity of the spine, history of a bleeding disorder c. Suspected elevated intracranial pressure, Arnold Chiari malformation or mass lesion d. Use of the anticoagulant medications such as but not limited to warfarin, rivaroxaban, dabigatran 18. Deemed ineligible by the Site PI for any other reason
Study design
Enrollment target: 850 participants
Age groups: adult
Timeline
Starts: 2018-04-30
Estimated completion: 2026-05-31
Last updated: 2026-02-18
Interventions
Drug: FlortaucipirDrug: FlorbetabenDrug: Fluorodeoxyglucose
Primary outcomes
  • 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))
Sponsor
Indiana University · other
With: Alzheimer's Therapeutic Research Institute, National Institute on Aging (NIA), Alzheimer's Association
Contacts & investigators
ContactIU LEADS Team · contact · iuLEADS@iupui.edu · 317-963-7436
InvestigatorLiana Apostolova, MD · principal_investigator, Indiana University
All locations (23)
Banner Sun Health Research InstituteCompleted
Sun City, Arizona, United States
University of California, Los AngelesRecruiting
Los Angeles, California, United States
Stanford UniversityRecruiting
Palo Alto, California, United States
University of California, San FranciscoRecruiting
San Francisco, California, United States
Georgetown UniversityCompleted
Washington D.C., District of Columbia, United States
Mayo Clinic, JacksonvilleRecruiting
Jacksonville, Florida, United States
Wien CenterCompleted
Miami Beach, Florida, United States
Emory UniversityRecruiting
Atlanta, Georgia, United States
Northwestern UniversityCompleted
Chicago, Illinois, United States
Indiana UniversityRecruiting
Indianapolis, Indiana, United States
Johns Hopkins UniversityRecruiting
Baltimore, Maryland, United States
Massachusetts General HospitalRecruiting
Boston, Massachusetts, United States
Mayo Clinic, RochesterRecruiting
Rochester, Minnesota, United States
Washington University, St. LouisRecruiting
St Louis, Missouri, United States
Columbia UniversityRecruiting
New York, New York, United States
University of PennsylvaniaRecruiting
Philadelphia, Pennsylvania, United States
Butler HospitalRecruiting
Providence, Rhode Island, United States
Houston Methodist HospitalRecruiting
Houston, Texas, United States
FleniRecruiting
Buenos Aires, Argentina
Amsterdam UMCNot Yet Recruiting
Amsterdam, Netherlands
Hospital de la Santa Creu i Sant PauRecruiting
Barcelona, Spain
Lund UniversityNot Yet Recruiting
Malmo, Sweden
University College LondonNot Yet Recruiting
London, United Kingdom
Longitudinal Early-onset Alzheimer's Disease Study Protocol · TrialPath