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 r
| Condition(s) | Gaucher Disease |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| 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. |
| Who can participate | Inclusion Criteria: * Diagnosis of Gaucher Disease Exclusion Criteria: * Not meeting Inclusion criteria |
| Sex | All |
| Lead sponsor | Duke University |
| Locations | Durham, North Carolina, United States |
| Start date | 2017-06-12 |
| NCT ID | NCT03190837 |
| Official listing | https://clinicaltrials.gov/study/NCT03190837 |