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A Long-term Follow-up Study of Gaucher Disease

NCT03190837 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2025-09-22

No results posted yet for this study

Summary

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), and c) identify potential long-term complications of this disorder. These aims will be accomplished through long-term record review of individuals with all three types of Gaucher disease.

Conditions

  • Gaucher Disease

Sponsors & Collaborators

Principal Investigators

  • Priya Kishnani, MD · Duke University

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-06-12
Primary Completion
2027-01-31
Completion
2027-01-31

Countries

  • United States

Study Locations

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Entities

Companies

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 NCT03190837 on ClinicalTrials.gov