clinical trial · NCT03643562
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Niemann-Pick Type C DiseaseAdrabetadex
brief summary
This study was amended from expanded 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. 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.
started
Jun 18, 2018
primary completion
Nov 2, 2021
completion
Nov 2, 2021
last updated
Jan 17, 2024
official title
Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol