Long-term follow-up for Gaucher disease
A Long-term Follow-up Study of Gaucher Disease
Duke University · NCT03190837
This study looks at how well enzyme replacement therapy and substrate reduction therapy work for people with Gaucher disease over the long term by tracking their health records.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Sex | All |
| Sponsor | Duke University (other) |
| Locations | 1 site (Durham, North Carolina) |
| Trial ID | NCT03190837 on ClinicalTrials.gov |
What this trial studies
This study aims to understand the long-term natural history of Gaucher disease by evaluating the efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). It involves systematic monitoring of health records from patients diagnosed with all three types of Gaucher disease. Health information will be collected from medical records at Duke University and from treating physicians outside the institution. The principal investigator, Dr. Kishnani, is a recognized expert in the field and will oversee the annual monitoring of patient health status without altering their standard care.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with any type of Gaucher disease.
Not a fit: Patients who do not have a diagnosis of Gaucher disease will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term management and treatment outcomes for patients with Gaucher disease.
How similar studies have performed: Other studies have shown success in understanding the natural history and treatment efficacy in Gaucher disease, making this approach both relevant and necessary.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of Gaucher Disease Exclusion Criteria: * Not meeting Inclusion criteria
Where this trial is running
Durham, North Carolina
- Duke University Medical Center — Durham, North Carolina, United States (RECRUITING)
Study contacts
- Principal investigator: Priya Kishnani, MD — Duke University
- Study coordinator: Janet Blount
- Email: janet.blount@duke.edu
- Phone: 919-681-7962
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Gaucher Disease, gaucher, gaucher disease