Drug Repurposing: Phase 3 trials (Milestone 7.E)
Timeline Start - End2021 - 2025
Research Implementation AreaDrug Repurposing and Combination Therapy Development
Initiate at least three Phase 3 trials with repurposed drugs or drug combinations. Of these at least one trial will be in an asymptomatic at-risk population (e.g., mutation carriers, APOE4 positive, Down Syndrome, high amyloid burden, etc.) and at least one for individuals with advanced disease. Of the trials initiated in patients with advanced disease, at least one trial will target the neuropsychiatric symptoms of Alzheimer’s disease and related dementias.
- Comprehensive success/failure analyses of data from at least three Phase 3 trials.
Summary of Key Accomplishments
Two late-stage trials of repurposed drug candidates have been supported. A Phase 3 trial testing a low-dose formulation of levetiracetam (an anti-seizure drug) for treating the symptoms and progression of mild cognitive impairment due to AD is underway; its anticipated completion is by the end of 2022. A Phase 2/3 trial testing the diabetes drug metformin for preventing cognitive decline among persons with mild cognitive impairment has also been initiated; its anticipated completion is in 2025.
The key accomplishments summary is current as of March 2022.
- PAR-16-364: Late Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01)
- PAR-18-028: Late Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01)
- PAR-18-878: Late Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01)
- PAR-18-513: Alzheimer's Clinical Trials Consortium (ACTC) Clinical Trials (R01)
- PAR-21-359: Early and Late Stage Clinical Trials for the Spectrum of Alzheimer’s Disease/Alzheimer’s Related Dementias and Age-Related Cognitive Decline (R01 Clinical Trial Optional)
Research Programs and Resources
- Alzheimer’s Clinical Trials Consortium (ACTC)
- NIA Ongoing Alzheimer’s and Related Clinical Trials and Studies
- A Phase 3 Pivotal Trial of AGB101 to Slow Progression in MCI due to Alzheimer’s Disease
- Therapeutic Effect of Intranasal Insulin on Cognition, Function, and AD Biomarkers
- Metformin in Alzheimer's dementia Prevention (MAP)