Trials / Completed
CompletedNCT02435030
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
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
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 35 (actual)
- Sponsor
- ZevraDenmark · Industry
- Sex
- All
- Age
- 2 Years – 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Timeline
- Start date
- 2015-09-01
- Primary completion
- 2017-05-01
- Completion
- 2017-05-01
- First posted
- 2015-05-06
- Last updated
- 2017-05-18
Locations
16 sites across 8 countries: Denmark, France, Germany, Italy, Poland, Spain, Switzerland, United Kingdom
Source: ClinicalTrials.gov record NCT02435030. Inclusion in this directory is not an endorsement.