Cyclo Therapeutics has completed patient enrolment in the Phase I/II clinical trial of intravenous Trappsol Cyclo in patients with Niemann-Pick Disease Type C1 (NPC1).
Trappsol Cyclo is a cyclodextrin-based product that received orphan drug designation in the US and Europe. It is currently undergoing three clinical trials in NPC patients.
NPC is a rare, genetic disease that impacts one in 100,000 newborns worldwide. It develops because of a defect in the NPC1 protein that is associated with cholesterol processing in the cell.
The disease leads to symptoms in multiple organs, including the brain, liver, spleen and lungs. It currently lacks approved drug therapies in the US and only one treatment is approved in Europe.
During the randomised, double-blind trial, Trappsol Cyclo will be assessed in 12 patients aged two years and above. Participants will be administered 24 1,500mg/kg, 2,000mg/kg, or 2,500mg/kg doses of the drug.
The trial will monitor adverse events, cholesterol metabolism markers after drug administration, and symptomatic changes with an NPC severity scoring tool, among other measures.
Initial study findings indicate an encouraging safety profile and a temporal association of the drug administration with cholesterol clearance from cells.
Data also revealed that the presence of the drug in the cerebrospinal fluid was linked with a decrease in tau, a neuron-specific biomarker linked to neuronal degeneration in NPC.
Trappsol Cyclo led to improvements in neurologic symptoms in the initial two patients who completed the trial.
Cyclo Therapeutics chairman and CEO N Scott Fine said: “Today’s ‘Last-Patient-In’ announcement is a major milestone for our company and the NPC community. It completes another important step in our development and registration strategies for Trappsol Cyclo to treat NPC, a disease which causes so much suffering for the patients and their families.”
In October last year, the company concluded enrolment in a Phase I trial of Trappsol Cyclo in NPC.