A Long-term Follow-up Study of Gaucher Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 10/24/2018 |
| Start Date: | June 12, 2017 |
| End Date: | January 2027 |
| Contact: | Lauren Bailey |
| Email: | lauren.bailey@duke.edu |
| Phone: | 919-613-0948 |
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.
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.
The study aims (aims a-c) will be accomplished by the systematic monitoring of health
records. Health information will be extracted from medical records for patients followed at
Duke. Medical records will be requested from treating physicians outside of Duke. The PI,
(Dr. Kishnani) a recognized expert in Gaucher disease, is a consultant to the treating
physician of virtually all subjects in this study. Subject health status will be monitored at
least on an annual basis and as regularly as the medical information is obtained from the
treating physician. Enrollment in this study will not alter the standard of care treatment
for patients with Gaucher disease.
records. Health information will be extracted from medical records for patients followed at
Duke. Medical records will be requested from treating physicians outside of Duke. The PI,
(Dr. Kishnani) a recognized expert in Gaucher disease, is a consultant to the treating
physician of virtually all subjects in this study. Subject health status will be monitored at
least on an annual basis and as regularly as the medical information is obtained from the
treating physician. Enrollment in this study will not alter the standard of care treatment
for patients with Gaucher disease.
Inclusion Criteria:
- Diagnosis of Gaucher Disease
Exclusion Criteria:
- Not meeting Inclusion criteria
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