📋 Research Plan
Alzheimer's diseaseTREM2$986,50018-24 monthsN = 300
Assay
Phase 2 RCT comparing TREM2 modulator vs. placebo in N=300 early AD patients, primary endpoint: change from baseline on composite cognitive score at 18 months. Sample size powered at 80% for effect size=0.45, alpha=0.05.
Primary endpoint
Composite cognitive score (ADAS-Cog13 + ADCS-ADL)
Kill criteria
- TREM2 loss-of-function variants show no AD risk modulation in replication cohort (OR=1.0, p>0.05)
- Microglial TREM2 knockout in 5xFAD mice fails to alter amyloid plaque burden
- TREM2 agonist fails to reduce tau pathology in P301S tauopathy model
Power: {'test': 'two-sided t-test', 'alpha': 0.05, 'power': 0.8, 'effect_size': 0.45}