China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Study Overview
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Brief Summary
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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.

Detailed Description
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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

Official Title
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A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease

Conditions
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Fabry Disease

Intervention / Treatment
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* Drug: Agalsidase beta


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant must be 8 years of age or older, at the time of signing the informed consent Participants naive to agalsidase beta and agalsidase alpha Chinese participants diagnosed with Fabry disease and with documented plasma or leukocyte αGAL activity deficient below laboratory's reference range, and/or documented diagnosis by genotyping Participants must have one or more symptoms and signs consistent with manifestations of Fabry disease (not limited to neuropathic pain, chronic kidney disease, hypertrophic cardiomyopathy, cardiac rhythm disturbances, cerebrovascular involvement, cornea verticillata, angiokeratoma, gastrointestinal symptoms, hypo- or anhydrosis) A female participant is eligible to participate if she is not pregnant or breastfeeding and use an acceptable contraceptive method Participants and/or participant's legal representative capable of giving signed informed consent. Exclusion Criteria: The participant has undergone kidney transplantation. The participant has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) in the opinion of the Investigator, would preclude participation in the trial. Received an investigational drug, or device, other than Fabrazyme, within 30 days of anticipated IMPs administration or 5 half-lives of the previous investigational drug, whichever is longer. The patient has current evidence of kidney failure or renal insufficiency, as defined by eGFR <30 mL/min/1.73 m2. Individuals who have life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients included. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Ages Eligible for Study
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Minimum Age: 8 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Agalsidase beta<br>Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion | Drug: Agalsidase beta<br>* Powder for concentration into a solution Intravenous (IV) infusion<br>* Other names: GZ419828 Fabrazyme;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Baseline to week 50 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The absolute changes of plasma globotriaosylsphingosine (lyso-GL3) |  | from baseline to Week 6, Week 12, Week 24 and Week 48 | 
| The percent changes of plasma lyso-GL3 |  | from baseline to Week 6, Week 12, Week 24 and Week 48 | 
| The absolute changes of plasma globotriaosylceramide (GL3) |  | from baseline to Week 6, Week 12, Week 24 and Week 48 | 
| The percent changes of plasma GL3 |  | 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 |  | at Week 6, Week 12, Week 24 and Week 48 | 
| The percentage of participants with abnormal plasma GL3 values per central lab reference range |  | 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 | 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) |  | 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) |  | from baseline to Week 12, Week 24, Week 36 and Week 48 |