Clinical Study

Testing a new drug called Trappsol® Cyclo in patients with Niemann-Pick Disease Type C1

Niemann-Pick Disease Type C1 (NPC1) is a disease that may cause problems in the brain and the body. This study will test a drug called Trappsol® Cyclo in children and adults with NPC1 to see if it helps treat their disease. Participants in this study will be randomized to be treated with Trappsol® Cyclo or a placebo in an IV infusion. The IV infusion will happen in the clinic every 2 weeks. Being in this study requires up to 100 study visits at the clinic. Later in the study, participants on the placebo can be switched to Trappsol® Cyclo.

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