Are large simple trials for dementia prevention possible?

Area of Science:

• Neuroscience and Public Health
• Clinical Trial Design

Background:

• Dementia prevention research requires novel strategies and agents.
• Large, simple cardiovascular trials offer a successful model for discovering treatments with moderate effects.
• Lessons from cardiovascular trial design can inform dementia prevention studies.

Purpose of the Study:

• To outline suitable populations, interventions, and outcomes for large simple trials in dementia prevention.
• To identify features necessary for maximizing the efficiency of dementia prevention trials.
• To address the challenges of participant size and follow-up duration in dementia prevention research.

Main Methods:

• Consideration of diverse populations, including those selected by age, clinical or genetic risk factors, or clinical presentation.
• Prioritization of functional and clinical outcomes, as well as dementia diagnoses, over cognitive scores and biomarker levels.
• Adaptation of large, simple trial designs, proven effective in cardiovascular research, for dementia prevention.

Main Results:

• Functional and clinical outcomes, including loss of function or dementia diagnoses, are meaningful measures for participants and can be assessed in large trials.
• Suitable populations can be identified through various demographic, clinical, and genetic criteria.
• Efficiency in large simple trials can be maximized by careful selection of populations, interventions, and outcomes.

Conclusions:

• Large simple trials are a viable and necessary approach for dementia prevention research.
• Participant and patient priorities for functional and clinical outcomes should guide trial design.
• Overcoming challenges in participant numbers and follow-up duration necessitates extensive collaboration among investigators, funders, and patient organizations.