Overview

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Status:
Active, not recruiting

Trial end date:
2021-10-01

Target enrollment:
0

Participant gender:
All

Summary

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.

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Mandos LLC
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company

Criteria

Inclusion criteria:

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

Exclusion criteria:

- None of the inclusion criteria are applicable