US-based biotechnology firm CTD Holdings has started patient enrolment in the Phase I/II clinical trial of Trappsol Cyclo to treat patients suffering from Niemann-Pick disease type C (NPC) in Europe.
Trappsol Cyclo is a parenteral grade of a molecule known as hydroxypropyl beta-cyclodextrin, which comprises seven glucopyranose units.
The Phase I/II trial is designed to assess the safety and efficacy of intravenous Trappsol Cyclo in 12 NPC patients aged two and above.
Trappsol Cyclo will be evaluated at 1,500mg/kg, 2,000mg/kg and 2,500mg/kg doses administered bi-weekly for 48 weeks.
While the first patient was recruited at Salford NHS Trust in the UK, additional clinical centres in the UK, Sweden and Italy will be included in the trial.
CTD chairman and CEO Scott Fine said: "Enrolment of the first patient at the Salford clinical site is a significant milestone for the company in the development of this treatment for a devastating disease.
“In addition, we anticipate enrolling the first patient in our US Phase I clinical trial of Trappsol Cyclo in the near future.”
Expected to report final results by the end of next year, the trial’s primary objective is determination of the injection's optimum dose for further study.
The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) previously granted fast-track designation and orphan drug designation for Trappsol Cyclo in NPC.
The data from administration of the injection to a total of 21 patients showed improvements in neurological symptoms, lung function or liver morphology, stabilisation of disease progression and no significant safety concerns.
Image: Niemann-Pick disease. Photo: courtesy of humpath.com.
