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Biomarkers: Incorporate biomarkers into Phase 3 trials (Milestone 9.E)


Timeline Start - End

2019 - 2024

Research Implementation Area


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

Success Criteria

Initiation of three Phase 3 (pivotal) drug trials using imaging and/or fluid biomarkers to select at risk subjects and/or for proof of target engagement.

Summary of Key Accomplishments

Eight AD Phase 3 trials for treatment/prevention have been enriched with imaging and/or fluid biomarkers. These biomarkers are used for selecting trial participants and to evaluate target engagement and treatment response. Across the eight trials, six new drug candidates (anti-amyloid) and two repurposed drugs (targeting metabolic and synaptic disfunction) are being tested/have been tested.

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 A4 trial is the first ongoing study to make the screening data and the biosamples available; one analysis of the screening data (genetic and brain imaging) revealed that non-Hispanic Blacks have lower levels of amyloid in the brain compared to non-Hispanic Whites.

The key accomplishments summary is current as of March 2022. 

Accomplishments/Implementation Activities

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