RecruitingPhase 3NCT06081062

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

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

Sponsor

ISU Abxis Co., Ltd.

Enrollment

24 participants

Start Date

Apr 14, 2023

Study Type

INTERVENTIONAL

Conditions

Fabry Disease

Summary

Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).

Eligibility

Min Age: 8 Years

Inclusion Criteria14

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:
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

Exclusion Criteria7

Patients who participated in other studies in which investigational products are administered within 30 days prior to the screening visit
Patients with chronic kidney disease stage 4 to 5 (CKD 4-5; see Section 16.1)
Patients who are currently on dialysis or have a history of kidney transplantation, or patients scheduled for dialysis at the time of screening, or waitlisted for kidney transplantation
Patients who have started angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) treatment within 4 weeks prior to the screening visit or whose dose has been changed
Patients who are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the clinical study
Patients with a history of HIV, hepatitis B/C or HIV antibodies, hepatitis B surface antigens, or hepatitis C antibodies
Patient whose medical, emotional, behavioral, or psychological conditions appear to interfere with compliance with the requirements of the clinical study according to the investigator's judgment