Multicenter and dose escalation phase 1 study to evaluate the safety and exploratory efficacy of GC1119 (recombinant human a-galactosidase A) for enzyme replacement therapy in Fabry disease patients
- Conditions
- Congenital malformations, deformations and chromosomal abnormalities
- Registration Number
- KCT0000597
- Lead Sponsor
- Green Cross
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 10
1) Subjects with a current diagnosis of Fabry's disease
(leukocyte a-gal activity of = 10 mnol/hr/ml and a mutation in a-galactosidase A gene)
2) Males = 16 years old
3) Subjects capable of performing this clinical trial in an appropriate manner
4) 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
5) Agreement to contraception during the study period
1) Serum creatinine > 2.5mg/dl
2) Subjects have a plan to kidney transplantation
3) Subjects have undergone kidney transplantation
4) Subjects are currently on dialysis
5) 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
6) Known life-threatening hypersensitivity(anaphylactic reaction) to a-galactosidase
7) 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
8) Known hypersensitivity to any of the ingredients of study drug(including excipients)
9) Subjects need the medication of prohibited drug
10) Alcoholism or drug addiction
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of adverse events
- Secondary Outcome Measures
Name Time Method Change and % change of Plasma GL-3 concentration;The ratio of subjects whose plasma GL-3 values are within reference range;Change and % change of urine GL-3 concentration;Change and % change of kidney function;Change and % change of heart size;Change of scores that are measured by pain questionnaire