Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).
The primary objective is to evaluate the efficacy of Fabagal compared to active comparator (Agalsidase beta). The secondary objectives will evaluate the safety, pharmacokinetics, and immunogenicity of Fabagal compared to active comparator (Agalsidase beta).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
24
1 mg/kg every 2 weeks for 12 months
1 mg/kg every 2 weeks for 12 months
Philippine General Hospital
Manila, Philippines
RECRUITINGSt.Luke's Medical Center
Manila, Philippines
RECRUITINGSeoul Asan Center
Seoul, Songpa-gu, South Korea
RECRUITINGEvaluate the efficacy of Fabagal compared with active comparator (Agalsidase beta)
The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (Agalsidase beta) in patients with Fabry disease The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (agalsidase beta) in patients with Fabry disease The primary endpoint is a comparison of the proportion of subjects in each group who achieve a score of Zero on the renal capillary endothelium histology. The score is graded on a scale from 0-3 (normal, mild, moderate, and severe). * 0: signified no visible inclusions * 1: signified multiple discrete lipid granules * 2: signified single or multiple aggregates of lipid granules * 3: signified aggregates of lipid granules either large enough or numerous enough to cause clear distortion of the luminal surface
Time frame: Screening visit (Visit 0) and Visit 25 (Day 336)
Change from baseline in GL-3 levels in renal cells after administration of 24 doses of Fabagal compared to active comparator
After administering Fabagal injection and active control group 24 times, changes in GL-3 levels in kidney cells compared to baseline will be confirmed.
Time frame: Screening visit (Visit 0) and Visit 25 (Day 336)
Change from baseline in GL-3/lyso Gb-3 concentration in urine and blood after administration of 24 doses of Fabagal compared to active comparator
Plasma and urine GL-3 are often elevated in the plasma of patients diagnosed with Fabry disease. After administering Fabagal injection and the active control group 24 times, the change in GL-3 and lyso Gb-3 concentrations in urine and blood compared to the baseline value will be confirmed.
Time frame: Screening visit (Visit 0), Visits 3, 5, 7, 9, and 11 (Days 28, 56, 84, 112, and 140), Visit 13 (Day 168), Visits 15, 17, 19, 21, and 23 (Days 196, 224, 252, 280, and 308) and Visit 25 (Day 336)
Change from baseline in renal function test values (estimated Glomerular Filtration Rate, eGFR) after administration
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Evaluated at Screening visit (Visit 0, Baseline), Visit 13 (Day 168) and Visit 25 (Day 336). eGFR is an estimation of the glomerular filtration rate of the renal (how much blood the kidneys are filtering).
Time frame: Screening visit (Visit 0), Visit 13 (Day 168), and Visit 25 (Day 336)
Change from baseline in pain score after administration of 24 doses of Fabagal compared to active comparator (Short Form McGill Pain Questionnaire-2)
The SF-MPQ-2 has 22 questions (6 on persistent pain, 6 on intermittent pain, 6 on neuropathic pain, and 4 on emotional pain) to be rated on an 11-point intensity scale (0 for not at all, to 10 for worst thinkable). Pain scores are presented as the mean of the ratings of all items.
Time frame: Visits 1, 13, and 25 (Days 0, 168, and 336)
Change from baseline in quality of life after administration of 24 doses of Fabagal compared to active comparator (SF-36v2 questionnaire)
The SF-36v2 consists of 36 questions(8-domain) to measure functional health and happiness from the patient's perspective. The 8 domains are physical functioning, role - physical, bodily pain, general health, vitality, social functioning, role - emotional, and mental health. The domain score is the sum of the weights of questions in each item. The score distribution ranges from 0 to 100, with a low score indicating a poor quality of life.
Time frame: Visits 1, 13, and 25 (Days 0, 168, and 336)