Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Status:
Active, not recruiting
Trial end date:
2021-11-01
Target enrollment:
Participant gender:
Summary
This study was amended from extended access to a clinical trial. Information will be
collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2
weeks.
Participants who were already taking adrabetadex will receive their stable dose. Participants
who have not ever taken it will start by receiving 400 mg.
The study design will be like the expanded access by Rush University, but for children at
least 4 years of age at screening.
Participants will receive treatment every 2 weeks until their doctor finds it does not help
them anymore, they withdraw, or the study is stopped for any reason. Participants will not
receive additional study treatment after their participation in this protocol.
Phase:
Phase 3
Details
Lead Sponsor:
Mandos LLC Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company