📋 Research Plan
alzheimerAPP$6,800,00036 monthsN = 300
Assay
Protocol: Oral administration of candidate compound (DP-084 or analog) twice daily + monthly CSF sampling.
Cohort: Early AD (MMSE 18-26), N=300, 2:1 randomization (active:placebo), 24-month treatment.
Compound: Small-molecule Aβ oligomerization inhibitor (MW < 500 Da) with transporter-mediated BBB crossing.
Controls: Placebo capsules, matched for size and appearance.
Readouts: (1) CSF Aβ oligomers (ELISA, primary), (2) PET amyloid (secondary), (3) CSF NfL, (4) cognition (ADAS-Cog13 + CDR-SB), (5) safety labs q3mo.
Sample size: 300 gives 90% power to detect 35% reduction in Aβ oligomers (effect size 0.5).
Primary endpoint
24-month change in CSF soluble Aβ oligomer concentration from baseline
Kill criteria
- Aβ oligomer reduction < 25% at any timepoint
- Cognitive decline not slowed (CDR-SB worsening > 2 points)
- Drug-related SAEs > 8%
- Exposure-response not established by 18 months
Power: {'test': 'mixed-effects model', 'alpha': 0.05, 'power': 0.9, 'effect_size': 0.5, 'expected_dropout': 0.25}