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

NCT02435030 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 35

Last updated 2017-05-18

No results posted yet for this study

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

  • Niemann-Pick Disease, Type C

Sponsors & Collaborators

  • ZevraDenmark

    lead INDUSTRY

Principal Investigators

  • Karl-Eugen Mengel · Villa Metabolica, Mainz, Germany

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-09-30
Primary Completion
2017-05-31
Completion
2017-05-31

Countries

  • Denmark
  • France
  • Germany
  • Italy
  • Poland
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02435030 on ClinicalTrials.gov