Sample size estimates for biomarker-based outcome measures in clinical trials in autosomal dominant Alzheimer's disease.

ABSTRACT:

Introduction

Alzheimer disease (AD)-modifying therapies are approved for treatment of early-symptomatic AD. Autosomal dominant AD (ADAD) provides a unique opportunity to test therapies in presymptomatic individuals.

Methods

Using data from the Dominantly Inherited Alzheimer Network (DIAN), sample sizes for clinical trials were estimated for various cognitive, imaging, and CSF outcomes. Sample sizes were computed for detecting a reduction of either absolute levels of AD-related pathology (amyloid, tau) or change over time in neurodegeneration (atrophy, hypometabolism, cognitive change).

Results

Biomarkers measuring amyloid and tau pathology had required sample sizes below 200 participants per arm (examples CSF Aβ42/40: 47[95%CI 25,104], cortical PIB 49[28,99], CSF p-ta

SUBMITTER: Cash DM 

PROVIDER: S-EPMC11601746 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature