Fabry Disease
Conditions
Brief summary
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.
Interventions
DRUGGC1119
biweekly, IV infusion
Sponsors
Green Cross Corporation
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects with a current diagnosis of Fabry's disease * Plasma α-gal activity of ≤ 1.5mnol/hr/ml and have a mutation in α-galactosidase A gene * Males ≥ 16 years old * Subjects capable of performing this clinical trial in an appropriate manner * Informed consent form voluntarily signed by the subject(or his legally acceptable representative if the subject is under 20 years old) to participation in the study * Agreement to contraception during the study period
Exclusion criteria
* Serum creatinine \> 2.5mg/dl * Subjects have a plan to kidney transplantation * Subjects have undergone kidney transplantation * Subjects are currently on dialysis * Subjects have a clinically significant organic disease(cardiovascular, hepatic, pulmonary, neurologic, or renal disease)that in the opinion of the investigator would preclude participation in the trial * Known life-threatening hypersensitivity(anaphylactic reaction) to α-galactosidase * Treatment with another investigational product within 30days from the administration of study drug dosing or plans to be treated with another investigational product during the study period * Known hypersensitivity to any of the ingredients of study drug(including excipients) * Subjects need the medication of prohibited drug * Alcoholism or drug addiction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events | 10weeks |
Secondary
| Measure | Time frame |
|---|---|
| The ratio of subjects whose plasma GL-3 values are within reference range | 10weeks |
| change and %change of urine GL-3 concentration | baseline and 10weeks |
| change and %change of kidney function | baseline and 10weeks |
| change and %change of Plasma GL-3 concentration | baseline and 10weeks |
| change and %change of heart size | baseline and 10weeks |
| change of results of cornial opacity examination | baseline and 10weeks |
| change of scores that are measured by pain questionnaire | baseline and 10weeks |
| change and %change of kidney size | baseline and 10weeks |
Countries
South Korea
Outcome results
None listed