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RECRUITING
NCT03190837
A Long-term Follow-up Study of Gaucher Disease
Sponsor: Duke University
View on ClinicalTrials.gov
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.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
200
Start Date
2017-06-12
Completion Date
2027-01
Last Updated
2025-09-22
Healthy Volunteers
Not specified
Conditions
Locations (1)
Duke University Medical Center
Durham, North Carolina, United States