Gaucher DiseaseRare disease
What this study is
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.
Study details
- Sponsor
- Duke University
- Conditions
- Gaucher Disease
- Enrolling
- 200 people
- Starts
- 2017-06-12
- Est. completion
- 2028-01
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex ALLHealthy volunteers Not acceptedYou may qualify if
- Diagnosis of Gaucher Disease
You can't join if
- Not meeting Inclusion criteria
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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1
Study locations (1)
1 openDuke University Medical Center
Durham, North Carolina, United States
Recruiting
Study team
Sponsor
Duke University
Study ID Pro00081246
Principal investigator
Priya Kishnani, MD
Duke University
Have questions? The contacts above can answer them before you apply.
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.