View clinical trials related to Niemann-Pick Disease, Type C1.
Filter by:The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.