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In the study at the National Institutes of Health (NIH), 14 patients with NPC-1 disease were enrolled. They were treated monthly with increasing dose of HPBCD starting with 50 mg/kg bw to 900 mg/kg. Biomarker studies were consistent with improved neuronal cholesterol homoeostasis and decreased neuronal pathology. While decreased progression of neurodegeneration (cognition and speech) was detected, no drug related serious adverse effects were observed. Mid-frequency to high-frequency hearing loss, an expected adverse event, was documented in all participants. When managed with hearing aids, this did not have an appreciable effect on daily communication.
 
Daniel S Ory, Elizabeth A Ottinger, Nicole Yanjanin Farhat, Kelly A King, Xuntian Jiang, Lisa Weissfeld, Elizabeth Berry-Kravis, Cristin D Davidson, Simona Bianconi, Lee Ann Keener, Ravichandran Rao, Ariane Soldatos, Rohini Sidhu, Kimberly A Walters, Xin Xu, Audrey Thurm, Beth Solomon, William J Pavan, Bernardus N Machielse, Mark Kao, Steven A Silber, John C McKew, Carmen C Brewer, Charles H Vite, Steven U Walkley, Christopher P Austin, Forbes D Porter:
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial.
The Lancet, 2017; DOI: 10.1016/S0140-6736(17)31465-4

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