Evaluate the Safety and Exploratory Efficacy of GC1119

Study Overview
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Brief Summary
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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.

Official Title
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Multicenter and Dose Escalation Phase 1 Study to Evaluate the Safety and Exploratory Efficacy of GC1119(Recombinant Human α-galactosidase A) for Enzyme Replacement Therapy in Fabry Disease Patients

Conditions
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Fabry Disease

Intervention / Treatment
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* Drug: GC1119


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: GC1119 0.5 mg/kg<br>0.5 mg/kg biweekly | Drug: GC1119<br>* biweekly, IV infusion<br>|
| Experimental: GC1119 1.0 mg/kg<br>1.0 mg/kg biweekly | Drug: GC1119<br>* biweekly, IV infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events |  | 10weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| change and %change of Plasma GL-3 concentration |  | baseline and 10weeks | 
| 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 kidney size |  | 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 |