Evaluate the Safety and Exploratory Efficacy of GC1119
- Registration Number
- NCT01653444
- Lead Sponsor
- Green Cross Corporation
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 8
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description GC1119 0.5 mg/kg GC1119 0.5 mg/kg biweekly GC1119 1.0 mg/kg GC1119 1.0 mg/kg biweekly
- Primary Outcome Measures
Name Time Method Incidence of adverse events 10weeks
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (3)
Asan Medical Center
🇰🇷Songpa-gu, Seoul, Korea, Republic of
Soon Cung Hyang University Hospital
🇰🇷Yongsan-gu, Seoul, Korea, Republic of
Ajou University School of Medicine
🇰🇷Yeongtong-gu, Suwon, Korea, Republic of