Cyclo Therapeutics has concluded enrolment in a Phase I clinical trial of Trappsol Cyclo in patients suffering from Niemann-Pick Disease Type C (NPC).
Trappsol Cyclo is a cyclodextrin-based product with orphan drug designation in the US and Europe. It is currently undergoing three clinical trials for the treatment of NPC.
NPC is a rare, genetic disease estimated to impact one in 100,000 live births worldwide. It develops because of a defect in the NPC protein that is associated with cholesterol processing in the cell.
The disease affects every cell in the body and 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.
For the randomised, double-blind Phase I trial, 12 patients aged 18 and above were enrolled at a single site in the US. Participants will be treated with seven intravenous doses of 1,500mg/kg or 2,500mg/kg.
The study will track various parameters, including adverse events and cholesterol metabolism markers after treatment. Symptomatic changes will be determined with an NPC severity scoring tool.
Initial results from the trial revealed a favourable safety profile. It found a temporal link of drug administration with cholesterol clearance from cells, drug in the cerebrospinal fluid after intravenous administration, and a decrease in tau, a biomarker of neuronal degeneration in NPC.
Cyclo Therapeutics chairman and CEO Scott Fine said: “Today’s ‘last-patient-in’ announcement is a significant 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 much suffering for the patients and their families.”
Results from the last patient in the Phase I trial are expected to be reported in February 2020.
The Phase I data and findings from a companion Phase I/II study in the EU and Israel are anticipated to support the regulatory filings.