This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.
Study participation for each patient will be total of 54 weeks which will include 4 weeks of screening, 48 weeks of treatment period and 2 weeks of post study treatment observation
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
Powder for concentration into a solution Intravenous (IV) infusion
Investigational Site Number :1560003
Beijing, China
Investigational Site Number :1560002
Beijing, China
Investigational Site Number :1560001
Shanghai, China
Investigational Site Number :1560004
Shanghai, China
Incidence of treatment-emergent adverse events (AEs)
Including TEAE, SAEs, and adverse events of special interest (AESIs) including infusion associated reactions (IARs) and change of clinical laboratory, vital signs and ECG
Time frame: Baseline to week 50
The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The percent changes of plasma lyso-GL3
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The absolute changes of plasma globotriaosylceramide (GL3)
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The percent changes of plasma GL3
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The number of participants with abnormal plasma GL3 values per central lab reference range
Time frame: at Week 6, Week 12, Week 24 and Week 48
The percentage of participants with abnormal plasma GL3 values per central lab reference range
Time frame: at Week 6, Week 12, Week 24 and Week 48
The change of Fabry disease symptoms
The change of Fabry disease symptoms assessment (improved, worsen or same): angiokeratoma, sweating, chronic abdominal pain, level of activity, exercise tolerance and heat tolerance, headache, tinnitus
Time frame: from baseline to Week 24 and Week 48
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Investigational Site Number :1560006
Taiyuan, China
Investigational Site Number :1560005
Wuhan, China
The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)
Time frame: from baseline to Week 12, Week 24, Week 36 and Week 48
The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)
Time frame: from baseline to Week 12, Week 24, Week 36 and Week 48