Posted by CTD Holdings, Inc., a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, reports initial results from its two clinical trials using the company’s proprietary formulation of hydroxypropyl beta cyclodextrins (HPBCDs), Trappsol® Cyclo™, intravenously in patients with Niemann-Pick disease type C.
Results were presented at two scientific conferences, WORLDSymposium (We’re Organizing Research on Lysosomal Diseases), held Feb. 4 – 8, 2019, Orlando, Florida and the 13th Annual Brains for Brain (B4B) symposium organized under the auspices of the European Task Force on Brain and Neurodegenerative Lysosomal Storage Diseases, January 24-26, 2019. CTD’s presentations were on February 6 and January 26, respectively. (Here are links to: WORLDSymposium Poster 1, WORLDSymposium Poster 2, and to the B4B presentation).
At both conferences, data were presented on the first four patients in the Phase I trial, “A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol® Cyclo™ (HP-β-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease,” (ClinicalTrials.gov NCT02939547) and the first four patients in the Phase I/II trial, “A Phase I/II Study to Evaluate the Safety and PK of IV Trappsol® Cyclo™ (HP-β-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Pharmacodynamic Effects of Treatment Upon Markers of Cholesterol Metabolism and Clinical Outcomes,”.
Further information is available at CTD website.