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Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

NCT02416661 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 299

Last updated 2021-05-28

No results posted yet for this study

Summary

International, multicenter, epidemiological study to demonstrate the correlation and predictive value of lyso-Gb1 concentration with the clinical severity of naïve, initially non-ERT/SRT Gaucher disease type 1 and during the study ERT/SRT-newly started Gaucher type 1 patients and to correlate lyso-Gb1 concentration with the clinical improvement of ERT or SRT treated Gaucher type 1 and the clinical course of non-treated patients based on GD-DS3

Conditions

  • Lysosomal Storage Diseases
  • Gaucher Disease
  • Sphingolipidoses

Sponsors & Collaborators

  • CENTOGENE GmbH Rostock

    lead INDUSTRY

Principal Investigators

  • Peter Bauer, M.D. · CENTOGENE GmbH Rostock

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-08-27
Primary Completion
2021-01-15
Completion
2021-01-15

Countries

  • Albania
  • Greece
  • India
  • Israel
  • Morocco
  • Pakistan
  • Spain

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 NCT02416661 on ClinicalTrials.gov