Life 4 Liam
Trappsol Cyclo
Cyclo Therapeutics’ cyclodextrin, given in a vein. It is in the Phase 3 TransportNPC study. Same broad idea as adrabetadex, different product and delivery.
How it works
Trappsol Cyclo is also hydroxypropyl-beta-cyclodextrin, made and dosed as its own product. It is given intravenously. The goal is the same kind of cholesterol binding, reached through the bloodstream rather than a lumbar puncture.
Cyclo Therapeutics is running the Phase 3 TransportNPC study. Dr. Caroline Hastings has been a principal investigator, including the newborn substudy. The study is how families access this product. It is not a prescription you can assume is available.
The other cyclodextrin product is adrabetadex . Do not treat them as the same medicine.
Who it is for
People enrolled in TransportNPC, including a newborn substudy. Early-treatment data in children under 3 have been presented. It is not an approved medicine, and it is not intrathecal adrabetadex.
What it does not do
It does not fix the NPC1 gene. Intravenous delivery is not the same as a spinal-fluid dose. Presented early-treatment data are not the same thing as FDA approval.
What this treatment does
Four short pictures of the idea. Play through, or tap a step. This is a teaching picture, not a medical simulation.
Step 1 of 4 · The problem
Cholesterol is stuck inside the lysosome
NPC1 cannot move it out. Trappsol is another attempt to bind that trapped cholesterol.
Official information
Educational only. Ask an NPC specialist what is right for a specific child or adult. Official sites and FDA pages are the source of prescribing information.