Evaluate the Safety and Exploratory Efficacy of GC1119

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01653444
Recruitment Status : Completed
First Posted : July 31, 2012
Last Update Posted : June 24, 2016
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: GC1119 Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : November 2012
Primary Completion Date : May 2014
Study Completion Date : October 2015

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: GC1119 0.5 mg/kg
0.5 mg/kg biweekly
Drug: GC1119
biweekly, IV infusion
Experimental: GC1119 1.0 mg/kg
1.0 mg/kg biweekly
Drug: GC1119
biweekly, IV infusion

Primary Outcome Measures :
  1. Incidence of adverse events [ Time Frame: 10weeks ]

Secondary Outcome Measures :
  1. change and %change of Plasma GL-3 concentration [ Time Frame: baseline and 10weeks ]
  2. The ratio of subjects whose plasma GL-3 values are within reference range [ Time Frame: 10weeks ]
  3. change and %change of urine GL-3 concentration [ Time Frame: baseline and 10weeks ]
  4. change and %change of kidney function [ Time Frame: baseline and 10weeks ]
  5. change and %change of kidney size [ Time Frame: baseline and 10weeks ]
  6. change and %change of heart size [ Time Frame: baseline and 10weeks ]
  7. change of results of cornial opacity examination [ Time Frame: baseline and 10weeks ]
  8. change of scores that are measured by pain questionnaire [ Time Frame: baseline and 10weeks ]

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01653444

Korea, Republic of
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
Sponsors and Collaborators
Green Cross Corporation

Responsible Party: Green Cross Corporation Identifier: NCT01653444     History of Changes
Other Study ID Numbers: GC1119_P1
First Posted: July 31, 2012    Key Record Dates
Last Update Posted: June 24, 2016
Last Verified: June 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders