Projektleiter:   Univ.-Prof. Dr. Julia Beatrice Hennermann, Univ.-Prof. Dr. Jörg Faber, Prof. Dr. Irene Krämer

A Phase IIb/III prospective, randomized, double-blind, sham-controlled trail of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) in subjects with neurologic manifestations of Niemann-Pick Type C1 (NPC1) Disease

Sponsor commissions the Clinical Center and the Investigator to carry out the Study in compliance with the procedures laid down in the test schedule / protocol. The services to be provided in connection with the Study contract by the Departments Paediatric Oncology, Paediatric Audiology and Pharmacy of the Clinical Center are described in supplements agreements
Laufzeit:  2016-2021