Validation study for lyso-Gb3 plasma concentration in healthy adult or child Japanese volunteers
- Conditions
- Fabry disease
- Registration Number
- JPRN-UMIN000016854
- Lead Sponsor
- Meiji Pharmaceutical University
- Brief Summary
Clin Exp Nephrol. doi: 10.1007/s10157-017-1525-3.(open access)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 100
Not provided
1)Volunteers who were diagnosed or suspected as Fabry disease 2)Volunteers with a history of receiving GLA enzyme replacement therapy (Replagal® or Fabrazyme®) 3)Volunteers who have previous or current disease or symptoms shown in Table 3 4)Volunteers who any their relatives (parents, brothers, sisters, sons, daughters, grandparents, or cousins) have Fabry disease 5)Volunteers who received amiodarone, chloroquine or hydroxychloroquine within 6 months before informed consent or plan to receive either of the drugs during the study 6)Volunteers who are considered unhealthy (having clinical issues for which any treatments or clinical observation are necessary) by the investigator at screening 7)Volunteers who have any histories of cardiovascular, liver, kidney, lung, endocrine, digestive, blood, respiratory, neuropsychiatric, or central nervous system diseases and are considered ineligible for the study by the investigator. 8)Volunteers who are otherwise considered ineligible for the study by the investigator
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method yso-Gb3 concentration in plasma
- Secondary Outcome Measures
Name Time Method GLA activity in plasma