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

Status Terminated

Clinical Trial Summary

Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant 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.

Clinical Trial Description

Participants in Part C will receive adrabetadex until the investigator considers adrabetadex to no longer be beneficial to the participant, or the development program is discontinued. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04958642
Study type	Interventional
Source	Mandos LLC
Contact
Status	Terminated
Phase	Phase 2/Phase 3
Start date	December 23, 2015
Completion date	April 11, 2022

View Details

See also
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