Niemann-Pick Disease, Type C1 research hub

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

This study is testing a new medicine called Trappsol Cyclo for Niemann-Pick Disease Type C1 (NPC1) in children and adults. It aims to see if the medicine is safe and effective compared to usual care. Some very young children can also take part in a separate part of the study.