Clinical Study of HPBCD in Patients with NPC Disease at Karolinska University Hospital

News 21 December, 2016
CTD Holdings, Inc. received the approval of Swedish Medical products Agency to conduct a Phase I/II clinical trial evaluating intravenous application of Trappsol® Cyclo™ in patients with Niemann Pick type C (NPC) disease, a rare and fatal illness manifested in neurodegenerative, genetic lisosomal lipid metabolism disorder [1]. In addition to this Phase I/II study is being conducted in Europe, a US Phase I study will be started soon. The first patients are expected to be enrolled in the first quarter of 2017.
Compared to the clinical trial conducted by NIH using intrathecally administered HPBCD of Vtess CTD expects good results by intravenous applications to avoid the more invasive route. To date, intravenous Trappsol® Cyclo™ has been administered to 21 NPC patients worldwide, some for more than five years, via multiple Compassionate Use Programs. During this time, multiple patients have shown marked improvements in neurological symptoms, lung function or liver morphology, or had stabilization of disease progression, with no significant, unexpected, safety concerns.
It is difficult for the parents of children in NPC disease to decide which clinical trial to join.