study started
estimated completion



Due to different study designs, the sponsor separated Part C into this separate registration (NCT number to be assigned), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events will be reported here. The primary and study completion dates are based on the anticipated last date safety data will be collected from Part C participants. This study is to find out how safe and effective adrabetadex is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.

Official Title

A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease


Participation in Part C is to continue until the investigator considers adrabetadex to be no longer beneficial to the participant, adrabetadex receives marketing authorization, or the development program is discontinued.


Niemann-Pick Disease, Type C Niemann-Pick Type C1 (NPC1) Disease neurologic disease gross motor dysfunction fine motor dysfunction dysphagia swallowing problems cognitive dysfunction gait abnormalities pediatrics Pick Disease of the Brain Niemann-Pick Diseases Niemann-Pick Disease, Type A Adrabetadex


You can join if…

One of the following is required for inclusion into VTS301 Part C:

  • Has agreed to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 weeks dosing regimen
  • The investigator has received prior written authorization from the sponsor for the participant to enter VTS301 Part C on an amended dose and/or regimen
  • Has received prior written authorization from Vtesse to enroll directly into Part C

You CAN'T join if...

  • None of the inclusion criteria are applicable


  • University of California San Francisco
    San Francisco California 94143-0780 United States
  • Children's Hospital of Orange County: CHOC Children's
    Orange California 92867 United States


in progress, not accepting new patients
Start Date
Completion Date
Mandos LLC
Phase 2/3
Study Type
Last Updated