Evaluate the Safety and Exploratory Efficacy of GC1119

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation
ClinicalTrials.gov Identifier:
NCT01653444
First received: July 24, 2012
Last updated: June 23, 2016
Last verified: June 2016
  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
Drug: GC1119
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Parallel 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:


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 ]

Enrollment: 8
Study Start Date: November 2012
Study Completion Date: October 2015
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01653444

Locations
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
  More Information

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: June 23, 2016
Health Authority: Korea: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: No

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
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular 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 July 26, 2016