📋 Research Plan
Alzheimer's diseaseLRP1$952,00018-24 monthsN = 300
Assay
Phase 2 RCT comparing LRP1 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=0.25, alpha=0.05.
Primary endpoint
Composite cognitive score (ADAS-Cog13 + ADCS-ADL)
Kill criteria
- LRP1-dependent tau uptake blockade shows no effect on tau propagation in human iPSC neurons
- LRP1 agonist fails to reduce tau seeding in 12-month observational study
- Blood-brain barrier LRP1-mediated clearance mechanism is not replicable in human studies
Power: {'test': 'two-sided t-test', 'alpha': 0.05, 'power': 0.8, 'effect_size': 0.25}