SUMMARY
# Lipid Droplet-Lysosome Axis Therapeutic Testing in Parkinson's Disease
## Background and Rationale
This clinical trial investigates a novel therapeutic approach targeting the lipid droplet-lysosome axis in Parkinson's disease (PD), addressing critical gaps in current treatment paradigms. PD pathogenesis involves complex interactions between protein aggregation, lipid metabolism dysfunction, and impaired autophagy-lysosomal clearance mechanisms. Recent evidence suggests that lipid droplets (LDs
METHODOLOGY NOTES
Phase 1 (Weeks 1-4): Screening and baseline assessments including comprehensive medical history, UPDRS evaluation, Montreal Cognitive Assessment (MoCA), DaTscan imaging, lumbar puncture for CSF collection, and blood sampling for lipid profiling and inflammatory markers. Participants undergo pharmacogenomic testing and medication washout period. Phase 2 (Weeks 5-8): Randomization to treatment (n=75) or placebo (n=75) groups. Treatment arm receives combination therapy: compound targeting acetyl-CoA carboxylase (200mg twice daily) plus lysosomal enhancer promoting TFEB nuclear translocation (150mg daily) plus standard levodopa therapy. Placebo group receives matching inert capsules plus levodopa. Weekly safety assessments monitor liver function, lipid panels, and adverse events. Phase 3 (Weeks 9-24): Continuation phase with monthly clinical evaluations including UPDRS Parts I-IV, Parkinson's Disease Questionnaire-39, and Epworth Sleepiness Scale. Biomarker sampling at weeks 12 and 24 incl