CTD Holdings has commenced patient dosing in a Phase I/II clinical trial of its intravenous drug candidate Trappsol Cyclo for the treatment of patients in Europe with Niemann-Pick disease type C (NPC).
Trappsol Cyclo is the parenteral grade of a molecule known as hydroxypropyl beta-cyclodextrin containing seven glucopyranose units.
The Phase I/II trial is designed to evaluate the safety and efficacy of the drug candidate in 12 NPC patients aged two and above at clinical sites in the UK, Sweden and Italy.
Patients will be administered with twice a week intravenous 1,500mg/kg, 2,000mg/kg and 2,500mg/kg Trappsol Cyclo injections over 48 weeks.
The first patient was dosed at Salford Royal NHS Foundation Trust in the UK by the trial’s coordinating investigator Dr Reena Sharma.
CTD Holdings chairman and CEO Scott Fine said: "Commencement of dosing of the first patient at the Salford clinical site is another significant milestone for the company in the development of our Trappsol Cyclo.
“CTD remains grateful for the continued support from the many patient families, researchers and clinicians, such as Dr Sharma, who have helped us reach this important milestone."
The final data from the Phase I/II trial is expected to be available by the end of next year.
Trappsol Cyclo has previously secured fast-track designation from the US Food and Drug Administration (FDA) and orphan drug designation for NPC from both the FDA and the European Medicines Agency (EMA).
The drug is being further studied as an intravenous formulation in a Phase I clinical trial for the treatment of NPC in the US.