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Biomarkers: Incorporate biomarkers into Phase 2 trials (Milestone 9.D)


Timeline Start - End

2017 - 2023

Research Implementation Area


Incorporate imaging and/or fluid biomarkers into Phase 2 (proof of concept) drug trials to provide proof of mechanism and/or evidence of target engagement as trials for 3-6 existing and 3-6 novel therapeutic targets are initiated.

Success Criteria

Enrich 3-6 Phase 2 (proof of concept) drug trials with imaging and/or fluid biomarkers for proof of target engagement and proof of clinical mechanisms.

Summary of Key Accomplishments

Eleven AD Phase 2 trials have been enriched with imaging and/or fluid biomarkers. These trials are testing three new drug candidates, six repurposed drugs, and two nutritional supplements. The embedding of imaging/fluid biomarkers trials will enable the investigators to evaluate whether the targeted disease process was affected by the treatment and led to beneficial change in AD/ADRD biomarkers. In addition, per NIA’s requirements, the data and biosamples from these trials will be made available to qualified investigators from the wider research community. This is key for enabling a comprehensive success/failure analysis and evaluating responsiveness to the treatment.

The key accomplishments summary is current as of March 2022. 

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