Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Inclusion Criteria: * Completion of study CTD-TCNPC-101 with no safety concerns at the final visit * Negative urine pregnancy test for females of child bearing potential * Patients must be legally resident in the USA with access to healthcare * Written, informed consent Exclusion Criteria: * Inability to comply with the proposed protocol assessments or any uncertainty about their ability to give meaningful, informed consent (legal guardian may give consent with subject assent) * Concurrent medical conditions representing a contraindication to any of the study medications * Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate (eGFR) \< 60mL/min/1.73m2 * Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or International Normalised Ratio (INR) \>1.8 * Male patients and female patients of childbearing potential who are not willing to use appropriate birth control (i.e. double barrier birth control) from enrollment until the follow-up visit