Received: 10 June 2019
Accepted: 21 September 2019
First Online: 21 October 2019
Ethics approval and consent to participate
: A data acquisition protocol was developed by the lead author (CH) and all investigators sought approval from local ethics or Institutional Review Boards. Case Report Forms (CRFs) were developed to capture patient characteristics including manifestations of disease, adverse events (AEs), details of drug administration, laboratory and clinical assessments. Informed consent was obtained from all of the subjects prior to the initiation of treatment with HPβCD in accordance to the local Institutional Review Boards and principles of ethical research according to the Declaration of Helsinki.
: Consent was obtained from the patient families by the investigators to participate in the data acquisition protocol with the intent to publish the results.
: <b>Caroline Hastings:</b> Principal Investigator, Phase I Study of the pharmacokinetics of Trappsol® Cyclo™ and effects on potential biomarkers of Niemann-Pick C1 (NPC1), ClinicalTrials.gov NCT02939547, CTD Holdings, Inc.; Senior clinical advisor and member of the Safety Review Committee EU Phase I/II Study of pharmacokinetics and preliminary efficacy of Trappsol® Cyclo™ in patients with Niemann-Pick C1 (NPC1), ClincialTrials.gov NCT02912793, CTD Holdings, Inc.; Co-Chair, Listening Circle, CTD Holdings, Inc.<b>Camilo Vieira:</b> None<b>Benny Liu:</b> Co-Principal Investigator, Phase I Study of the pharmacokinetics of Trappsol® Cyclo™ and effects on potential biomarkers of Niemann-Pick C1 (NPC1); ClinicalTrials.gov NCT02939547, CTD Holdings, Inc.; Scientific Advisory Board, CTD Holdings, Inc.<b>Cyrus Bascon:</b> Clinical research associate, Data Acquisition Study of Compassionate Use HPβCD, CTD Holdings, Inc.<b>Claire Gao:</b> None<b>Raymond Wang:</b> Local Site Principal Investigator, Study of VTS-270 (2-hydroxypropyl-beta-cyclodextrin) to treat Niemann-Pick Type C1 (NPC1) disease, ClinicalTrials.gov NCT02534844, Vtesse, Inc.; Scientific Advisory Board for NPC, Sucampo Pharmaceuticals, Inc.<b>Alicia Casey:</b> None<b>Sharon Hrynkow:</b> Chief Scientific Officer and Senior Vice President for Medical Affairs; Co-Chair, Listening Circle; Co-Chair, Scientific Advisory Board, CTD Holdings, Inc.