Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Status:
Active, not recruiting
Trial end date:
2021-10-01
Target enrollment:
Participant gender:
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
Details
Lead Sponsor:
Mandos LLC Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company