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Evaluate the Safety and Exploratory Efficacy of GC1119

This study is currently recruiting participants.
Verified January 2012 by Green Cross Corporation
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation
ClinicalTrials.gov Identifier:
NCT01653444
First received: July 24, 2012
Last updated: July 30, 2012
Last verified: January 2012
History of Changes
  Purpose

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 Intervention Phase
Fabry Disease
 Biological: GC1119, 0.5mg/kg
Biological: GC1119, 1.0mg/kg
 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Green Cross Corporation:

Primary Outcome Measures:
  • Incidence of adverse events [ Time Frame: 10weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • change and %change of Plasma GL-3 concentration [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]
  • The ratio of subjects whose plasma GL-3 values are within reference range [ Time Frame: 10weeks ] [ Designated as safety issue: No ]
  • change and %change of urine GL-3 concentration [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]
  • change and %change of kidney function [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]
  • change and %change of kidney size [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]
  • change and %change of heart size [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]
  • change of results of cornial opacity examination [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]
  • change of scores that are measured by pain questionnaire [ Time Frame: baseline and 10weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: July 2012
Estimated Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Test Drug
recombinant human α-galactosidase A
 Biological: GC1119, 0.5mg/kg
biweekly, 0.5mg/kg IV infusion
Biological: GC1119, 1.0mg/kg
biweekly, 1.0mg/kg IV infusion

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01653444

Contacts
Contact: Han Wook Yoo, M.D., Ph.D. 82-2-3010-3374 hwyoo@amc.seoul.kr

Locations
Korea, Republic of
Asan Medical Center Recruiting
Songpa-gu, Seoul, Korea, Republic of
Contact: Han Wook Yoo, M.D., Ph.D.     82-2-3010-3374     hwyoo@amc.seoul.kr    
Principal Investigator: Han Wook Yoo, M.D., Ph.D.            
Soon Cung Hyang University Hospital Not yet recruiting
Yongsan-gu, Seoul, Korea, Republic of
Contact: Dong Hwan Lee, M.D., Ph.D.     82-2-709-9341     ldh@schmc.ac.kr    
Principal Investigator: Dong Hwan Lee, M.D., Ph.D.            
Ajou University School of Medicine Not yet recruiting
Yeongtong-gu, Suwon, Korea, Republic of
Contact: Young Bae Sohn, M.D.     82-31-219-4447     ybsohn@ajou.ac.kr    
Principal Investigator: Young Bae Sohn, M.D.            
Sponsors and Collaborators
Green Cross Corporation
  More Information

No publications provided

Responsible Party: Green Cross Corporation
ClinicalTrials.gov Identifier: NCT01653444     History of Changes
Other Study ID Numbers: GC1119_P1
Study First Received: July 24, 2012
Last Updated: July 30, 2012
Health Authority: Korea: Food and Drug Administration

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on June 17, 2013