A Long-term Follow-up Study of Gaucher Disease
ConditionGaucher Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorDuke University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Diagnosis of Gaucher Disease
Disqualifiers
Not meeting Inclusion criteria
Trial population
We will be recruiting patients with Gaucher Disease of all ages, followed at Duke University Medical Center, or non-Duke patients who have contacted Duke and expressed interest in participating.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Document effects of Gaucher disease in different systems of body, including nervous system, liver, and spleen.
Use Gaucher patient's natural histories to understand effects of Gaucher Disease long term.
Time frame
10 yearsSecondary outcomes
1
Document adverse events subjects experience on enzyme replacement therapy
Use Gaucher patient's therapy history to understand effects of enzyme replacement therapy long term.
Time frame
10 years2
Document adverse events of subjects on substrate reduction therapy
Use Gaucher patient's therapy history to understand effects of substrate reduction therapy long term.
Time frame
10 years3
Document long-term complications in Gaucher Disease.
Use Gaucher patient's natural histories to understand long-term complications of Gaucher Disease.
Time frame
10 years4
Change in 36-Item Short Form Survey (SF-36) collected every 6 months/1 year.
We will use the SF-36 that will be collected every 6 months to 1 year to assess quality-of-life in Gaucher patients.
Time frame
10 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov