Evaluate the Safety and Exploratory Efficacy of GC1119

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

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.

Study Overview

• Status: Completed
• Conditions: Fabry Disease
• Intervention / Treatment: Drug: GC1119

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul
    • Songpa-gu, Seoul, Korea, Republic of
      • Asan Medical Center
    • Yongsan-gu, Seoul, Korea, Republic of
      • Soon Cung Hyang University Hospital
  • Suwon
    • Yeongtong-gu, Suwon, Korea, Republic of
      • Ajou University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events	10weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Green Cross Corporation

Study record dates

Study Major Dates

• Study Start: November 1, 2012
• Primary Completion (Actual): May 1, 2014
• Study Completion (Actual): October 1, 2015

Study Registration Dates

• First Submitted: July 24, 2012
• First Submitted That Met QC Criteria: July 30, 2012
• First Posted (Estimate): July 31, 2012

Study Record Updates

• Last Update Posted (Estimate): June 24, 2016
• Last Update Submitted That Met QC Criteria: June 23, 2016
• Last Verified: June 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Fabry Disease
• Other Study ID Numbers: GC1119_P1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO