Japan Fabry Research - 001
Not Applicable
Recruiting
- Conditions
- Fabry disease
- Registration Number
- JPRN-jRCT1090220293
- Lead Sponsor
- Kazuya Tsuboi
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 20
Inclusion Criteria
1) ERT treatment-naive patients with Fabry disease
2) Patients receiving treatment with agalsidase alfa 0.2 mg/kg every two weeks
Exclusion Criteria
Patients with E66Q amino acid substitution
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The parameters measured included globotriaosylceramide (Gb3), globotriaosylsphingosine (Lyso-Gb3), left ventricular mass index (LVMI), brain natriuretic peptide (BNP), high-sensitivity troponin I (hs-Trop I), estimated glomerular filtration rate (eGFR), and anti-agalsidase alfa IgG antibody formation.
- Secondary Outcome Measures
Name Time Method