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

Niemann-Pick Disease, Type C Clinical Trial

Official title:
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C in Order to Characterise the Individual Patient Disease Profile and Historic Signo-symptomatology Progression Pattern

Clinical Trial Summary

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02435030
Study type Observational
Source Orphazyme
Contact
Status Completed
Phase N/A
Start date September 2015
Completion date May 2017
View Details
See also
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Active, not recruiting NCT05758922 - Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease Phase 2
Completed NCT03910621 - Safety and Efficacy of Miglustat in Chinese NPC Patients Phase 4
Available NCT04316637 - Early Access Program With Arimoclomol in US Patients With NPC