Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial - Hastings, Caroline; Liu, Benny; Hurst, Bryan; Cox, Gerald F.; Hrynkow, Sharon (Molecular genetics and metabolism, 2022)
Keywords: 2-Hydroxypropyl-beta-cyclodextrin; Adult; Biomarkers; Central Nervous System - metabolism; Cholesterol - metabolism; Cyclodextrin; Humans; Lysosome; Niemann-Pick Disease Type C; Niemann-Pick Disease, Type A; Pharmacodynamics; Pharmacokinetics; Quality of Life; CRP; PP; UCSF; Lysosome; Niemann-Pick Disease Type C; CK-MB; ITT; ALT; CNS; INR; URI; AEs; ABR; LC-MS/MS; SD; t1/2; 17D-NPC-CSS; CSF; Cmax; Pharmacodynamics; AST; HPβCD; 24(S)-HC; PBMCs; GFAP; Cyclodextrin; NPC1; AUC0-; Tmax; Vss; SAEs; Pharmacokinetics; IND; TEAEs