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

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.

Timeline

Start

2015-12-23

Primary completion

2022-04-11

Completion

2022-04-11

Outcome

Outcome not reported

Stopped (Business): “Terminated by previous Sponsor decision”

Drugs

Indications