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Drug Repurposing: Phase 3 trials (Milestone 7.E)

In Progress

Timeline Start - End

2021 - 2025

Research Implementation Area

Drug 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.

Success Criteria

  • 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. 

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