A Phase 3 Randomized, Double-blinded, Active-controlled, Multicenter Trial to Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).
• Those who have been diagnosed with Fabry disease by genetic and alpha-galactosidase A enzyme tests and grouped by sex are as follows:
‣ Male: Those who have confirmed GLA mutation (variation of α-galactosidase A gene) by genetic testing, and whose activity of alpha-galactosidase A in leukocytes is 5% or less than the normal mean value
⁃ Female: Those who have confirmed GLA mutation by genetic testing, and whose alpha-galactosidase A is within the normal range or is deficient
• Age: Those who are aged 8 years or older
• Those who have at least one of the following symptoms and signs:
‣ Glomerular filtration rate decreased (Inclusion criteria: 2 or more cases of 30 ≦ eGFR \< 90 mL/min/1.73 m2 \[adjusted for age \>40\] \[including results within 6 months of the screening visit, but including results within 12 months for patients with a 60 ≦ eGFR \< 90 mL/min/1.73 m2\])
∙ Proteinuria that is equivalent to microalbuminuria or worse (Inclusion criteria: 2 or more cases of creatinine 30 mg/g in random urine at least 24 hours apart \[including results within 6 months of the screening visit\] or ≥30 mg of albuminuria in 24-hour urine)
• For 24 hr urinary protein extraction (\>4 mg/m2/hr) or for spot urinary protein/creatinine ratio (≥200 mg/g \[Cr\]) \*Pediatrics: Aged \<19 years
⁃ Abnormal left ventricular function as evidenced by MRI or echocardiography
∙ Left ventricular mass index (LVMI)\* \>115 g/m2 (male), \>95 g/m2 (female) or
‣ Left ventricular wall thickness \>12 mm (However, in the case of patients with hypertension, patients must have blood pressure treatment for at least 6 months prior to administration of the same drug) etc.
• Clinically significant arrhythmias and conduction disturbances, etc.
⁃ Stroke or transient ischemic attack, etc., as evidenced by objective testing
• Patients who have not previously received enzyme replacement therapy (ERT) or Chaperone therapy for treatment of Fabry disease
• Patients who voluntarily consented and signed the informed consent form
• Patients (female patients and partners of male patients who are of childbearing potential) who have agreed to use a medically appropriate method of contraception (intrauterine device, condoms, surgical methods such as vasectomy) during the clinical study