Clinical trial

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

ZevraDenmarkCompletedStarted Sep 1, 201535 enrolledDenmark, France, Germany, Italy, Poland, Spain, Switzerland, United KingdomNCT02435030

Abstract

This is a prospective non-therapeutic observational study in NP-C patients. The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical, imaging, biological(biomarkers) and quality of life data. Patients will be offered enrollment into a Phase II/III study on arimoclomol at the end of the study.

Topics & conditions

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LSD

Keywords

NP-Clysosomal storage disorderlysosomal storage diseaseNPC2NPC1Niemann-Pick Type CNiemann-Pick,ArimoclomolNiemann-Pick Disease, Type C

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