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Trappsol Cyclo
- Development stage
- Phase 4
- Lead developer
- Cyclo Therapeutics
- Modality
- Small Molecules, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
Trappsol Cyclo is a proprietary intravenous formulation of hydroxypropyl beta cyclodextrin (HP-β-CD) developed to treat Niemann-Pick disease type C1 (NPC1) and being explored for Alzheimer’s disease. HP-β-CD binds unesterified cholesterol and facilitates its mobilization and clearance from cells, addressing lysosomal cholesterol accumulation characteristic of NPC1; systemic IV dosing achieves peripheral and central exposure and has shown improvements or stabilization in clinical measures and biomarkers over 48 weeks in pediatric and adult NPC1 patients, with acceptable tolerability. The product has received Orphan Drug designation in the US and EU for NPC, US Fast Track and Rare Pediatric Disease designations, and is being studied in a pivotal Phase 3 trial (TransportNPC). The developer is Cyclo Therapeutics.
- Brand names
- Trappsol Cyclo
- Other names
- hydroxypropyl beta cyclodextrinHP-beta-cyclodextrinHP-β-CDhydroxypropyl-β-cyclodextrin
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