Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
Not Applicable
- Conditions
- Fabry disease
- Registration Number
- JPRN-UMIN000017152
- 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
Inclusion Criteria
Not provided
Exclusion Criteria
The following patients should not be enrolled to the study; Patients who are considered ineligible for the study by the investigator
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method As a diagnostic bio-marker; 1) Plasma concentration of lyso-Gb3 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34] and other analogs) 3) GLA activity in plasma As a therapeutic bio-marker;; 1) Plasma concentration of lyso-Gb3 and the change from baseline in the concentration after initiation of the treatment with Replagal 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34]) and other analogs) after initiation of the treatment with Replagal, and the change from baseline in the analog concentration after initiation of the treatment with Replagal
- Secondary Outcome Measures
Name Time Method