A Long-term Follow-up Study of Gaucher Disease
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|ClinicalTrials.gov Identifier: NCT03190837|
Recruitment Status : Recruiting
First Posted : June 19, 2017
Last Update Posted : October 10, 2022
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|Condition or disease|
|Study Type :||Observational|
|Estimated Enrollment :||200 participants|
|Official Title:||A Long-term Follow-up Study of Gaucher Disease|
|Actual Study Start Date :||June 12, 2017|
|Estimated Primary Completion Date :||January 2027|
|Estimated Study Completion Date :||January 2027|
- Document effects of Gaucher disease in different systems of body, including nervous system, liver, and spleen. [ Time Frame: 10 years ]Use Gaucher patient's natural histories to understand effects of Gaucher Disease long term.
- Document adverse events subjects experience on enzyme replacement therapy [ Time Frame: 10 years ]Use Gaucher patient's therapy history to understand effects of enzyme replacement therapy long term.
- Document adverse events of subjects on substrate reduction therapy [ Time Frame: 10 years ]Use Gaucher patient's therapy history to understand effects of substrate reduction therapy long term.
- Document long-term complications in Gaucher Disease. [ Time Frame: 10 years ]Use Gaucher patient's natural histories to understand long-term complications of Gaucher Disease.
- Change in 36-Item Short Form Survey (SF-36) collected every 6 months/1 year. [ Time Frame: 10 years ]We will use the SF-36 that will be collected every 6 months to 1 year to assess quality-of-life in Gaucher patients.
- Change in Small Fiber Neuropathy Screening List (SFNSL) collected every 6 months/1 year. [ Time Frame: 10 years ]The investigators will use the Small Fiber Neuropathy Screening List every 6 months to 1 year to monitor small fiber neuropathy symptoms in Gaucher patients.
- Document number of subjects experiencing neurological symptoms related to Gaucher, by using Neurological Follow-up exam [ Time Frame: 10 years ]The investigators will use the Neurological follow-up exam, that will be performed at return visit and every 6 months to 1 year afterward, to screen patients for neurological symptoms related to Gaucher Disease.
- Change in Parkinson's checklist collected every 6 months/1 year. [ Time Frame: 10 years ]The investigators will use the Parkinson's checklist that will be collected every 6 months to 1 year to screen patients for Parkinson's symptoms potentially related to Gaucher Disease.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||Child, Adult, Older Adult|
|Sexes Eligible for Study:||All|
|Sampling Method:||Non-Probability Sample|
- Diagnosis of Gaucher Disease
- Not meeting Inclusion criteria
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03190837
|Contact: Janet Blountemail@example.com|
|United States, North Carolina|
|Duke University Medical Center||Recruiting|
|Durham, North Carolina, United States, 27710|
|Principal Investigator:||Priya Kishnani, MD||Duke University|
|Responsible Party:||Duke University|
|Other Study ID Numbers:||
|First Posted:||June 19, 2017 Key Record Dates|
|Last Update Posted:||October 10, 2022|
|Last Verified:||September 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
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