Taking people in United StatesNo phase
Long-term Follow-up of People with Gaucher Disease
Follows people with Gaucher disease to study its long-term course and treatments.
Plain-language title generated from the registry record
Registry title: A Long-term Follow-up Study of Gaucher Disease
Key facts
- Where
- Durham, United StatesSee the site
Where in United States
- Duke University Medical CenterDurham, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Duke University
- Trial code
- NCT03190837
- Record updated
- 3 September 2026
- Original description
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.Read full original descriptionShow less
A Long-term Follow-up Study of Gaucher Disease
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.
Inclusion: Diagnosis of Gaucher Disease.
Exclusion: Not meeting Inclusion criteria.