Long-term Follow-up Study of Gaucher Disease
This observational study is looking at the long-term effects of Gaucher disease on different body systems, including the nervous system, liver, and spleen. Researchers also want to understand how well treatments like enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) work over time, and to identify any long-term problems that might come from the disease. You can join this study if you have a diagnosis of Gaucher disease. The study will track these effects over 10 years by reviewing your medical records. The current recruitment status is unclear.
- Study design
- This is an observational study aiming to enroll 200 participants. It involves reviewing health records over a long period.
- What's involved
- Your health status will be monitored at least once a year by reviewing your medical records. This study will not change your standard medical care.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 10 years to document the effects of Gaucher disease.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Long-term Follow-up Study of Gaucher Disease
At a glance
Conditions
NCT03190837
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Duke University Medical Center
Durham, North Carolinano site contact published
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Priya Kishnani, MD · PRINCIPAL_INVESTIGATOR · Duke University
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
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Inclusion
What this trial measures
- Document effects of Gaucher disease in different systems of body, including nervous system, liver, and spleen.10 years
Use Gaucher patient's natural histories to understand effects of Gaucher Disease long term.