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

Green Cross Corporation3 sites in 1 country8 target enrollmentNovember 2012

ConditionsFabry Disease

InterventionsGC1119

DrugsGC1119

Overview

Phase: Phase 1
Intervention: GC1119
Conditions: Fabry Disease
Sponsor: Green Cross Corporation
Enrollment: 8
Locations: 3
Primary Endpoint: Incidence of adverse events
Status: Completed
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

November 2012

End Date

October 2015

Last Updated

9 years ago

Study Type

Interventional

Study Design

Parallel

Sex

Male

Investigators

Sponsor

Green Cross Corporation

Responsible Party

Sponsor

Eligibility Criteria

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

Arms & Interventions

GC1119 0.5 mg/kg

0.5 mg/kg biweekly

Intervention: GC1119

GC1119 1.0 mg/kg

1.0 mg/kg biweekly

Intervention: GC1119

Outcomes

Primary Outcomes

Incidence of adverse events

Time Frame: 10weeks

Secondary Outcomes

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)