A Phase 4 interventional study of Agalsidase beta in Fabry Disease, sponsored by Genzyme, a Sanofi Company. Completed at 6 sites in China. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2025-09-11.
Sponsored by Genzyme, a Sanofi Company · Phase 4, Interventional, and Treatment
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
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
This study's enrollment of 22 is close to the median of 22 across 105 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Genzyme, a Sanofi Company is the lead sponsor of 303 studies on the registry; 5 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion
Drug: Agalsidase beta
Powder for concentration into a solution Intravenous (IV) infusion
Also known as: GZ419828 Fabrazyme
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
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
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
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Genzyme, a Sanofi Company