CTD Holdings reports launch of US Phase I clinical study of Trappsol Cyclo in Niemann-Pick Disease Type C patients.
M2 PHARMA-March 24, 2017-CTD Holdings reports launch of US Phase I clinical study of Trappsol Cyclo in Niemann-Pick Disease Type C patients
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Biotechnology company CTD Holdings Inc (Other OTC:CTDH) reported on Thursday that it has started enrolling patients under its Phase I clinical study in the US to evaluate the intravenous administration of Trappsol Cyclo in patients with Niemann-Pick Disease Type C (NPC) at the Children's Hospital & Research Center Oakland.
Niemann-Pick Disease Type C (NPC) is reportedly a rare and fatal genetic disease that impacts the brain, lung, liver, spleen and other organs. In NPC patients, cholesterol accumulates abnormally in the body cells.
Dr Caroline Hastings, Pediatric Hematologist Oncologist at UCSF Benioff Children's Hospital Oakland, is the principal investigator for the US study. The US clinical study will enrol 12 patients.
The company stated that the intravenous Trappsol Cyclo has been administered to 21 NPC patients worldwide to date via compassionate use programmes. The data have demonstrated that multiple patients have shown marked improvements in neurological symptoms, lung function or liver morphology, or had stabilization of disease progression, with no significant safety concerns.
According to the company, Trappsol Cyclo is a parenteral grade of hydroxypropyl beta cyclodextrin, a donut-shaped molecule comprised of seven glucopyranose units. Its hydrophilic exterior allows it to move easily through the body and its inner hydrophobic cavity allows it to capture and hold certain types of molecules, including cholesterol. It has received Orphan Drug designation from the US Food and Drug Administration and the European Medicines Agency.
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|Date:||Mar 24, 2017|
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