The purpose of this study is to evaluate the safety and exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
8
biweekly, IV infusion
Asan Medical Center
Songpa-gu, Seoul, South Korea
Soon Cung Hyang University Hospital
Yongsan-gu, Seoul, South Korea
Ajou University School of Medicine
Yeongtong-gu, Suwon, South Korea
Incidence of adverse events
Time frame: 10weeks
change and %change of Plasma GL-3 concentration
Time frame: baseline and 10weeks
The ratio of subjects whose plasma GL-3 values are within reference range
Time frame: 10weeks
change and %change of urine GL-3 concentration
Time frame: baseline and 10weeks
change and %change of kidney function
Time frame: baseline and 10weeks
change and %change of kidney size
Time frame: baseline and 10weeks
change and %change of heart size
Time frame: baseline and 10weeks
change of results of cornial opacity examination
Time frame: baseline and 10weeks
change of scores that are measured by pain questionnaire
Time frame: baseline and 10weeks
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