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Evaluate the Safety and Exploratory Efficacy of GC1119

Primary Purpose

Fabry Disease

Status

Completed

Phase

Phase 1

Locations

Korea, Republic of

Study Type

Interventional

Intervention

GC1119

About this trial

This is an interventional treatment trial for Fabry Disease

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

Asan Medical Center
Soon Cung Hyang University Hospital
Ajou University School of Medicine

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

GC1119 0.5 mg/kg

GC1119 1.0 mg/kg

Arm Description

0.5 mg/kg biweekly

1.0 mg/kg biweekly

Outcomes

Primary Outcome Measures

Incidence of adverse events

Secondary Outcome Measures

change and %change of Plasma GL-3 concentration

The ratio of subjects whose plasma GL-3 values are within reference range

change and %change of urine GL-3 concentration

change and %change of kidney function

change and %change of kidney size

change and %change of heart size

change of results of cornial opacity examination

change of scores that are measured by pain questionnaire

Full Information

NCT ID

NCT01653444

First Posted

July 24, 2012

Last Updated

June 23, 2016

Sponsor

Green Cross Corporation

1. Study Identification

Unique Protocol Identification Number

NCT01653444

Brief Title

Evaluate the Safety and Exploratory Efficacy of GC1119

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 Type

Interventional

2. Study Status

Record Verification Date

June 2016

Overall Recruitment Status

Completed

Study Start Date

November 2012 (undefined)

Primary Completion Date

May 2014 (Actual)

Study Completion Date

October 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Green Cross Corporation

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Fabry Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

8 (Actual)

8. Arms, Groups, and Interventions

Arm Title

GC1119 0.5 mg/kg

Arm Type

Experimental

Arm Description

0.5 mg/kg biweekly

Arm Title

GC1119 1.0 mg/kg

Arm Type

Experimental

Arm Description

1.0 mg/kg biweekly

Intervention Type

Drug

Intervention Name(s)

GC1119

Intervention Description

biweekly, IV infusion

Primary Outcome Measure Information:

Title

Incidence of adverse events

Time Frame

10weeks

Secondary Outcome Measure Information:

Title

change and %change of Plasma GL-3 concentration

Time Frame

baseline and 10weeks

Title

The ratio of subjects whose plasma GL-3 values are within reference range

Time Frame

10weeks

Title

change and %change of urine GL-3 concentration

Time Frame

baseline and 10weeks

Title

change and %change of kidney function

Time Frame

baseline and 10weeks

Title

change and %change of kidney size

Time Frame

baseline and 10weeks

Title

change and %change of heart size

Time Frame

baseline and 10weeks

Title

change of results of cornial opacity examination

Time Frame

baseline and 10weeks

Title

change of scores that are measured by pain questionnaire

Time Frame

baseline and 10weeks

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

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

Facility Information:

Facility Name

Asan Medical Center

City

Songpa-gu

State/Province

Seoul

Country

Korea, Republic of

Facility Name

Soon Cung Hyang University Hospital

City

Yongsan-gu

State/Province

Seoul

Country

Korea, Republic of

Facility Name

Ajou University School of Medicine

City

Yeongtong-gu

State/Province

Suwon

Country

Korea, Republic of

12. IPD Sharing Statement

Plan to Share IPD

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Evaluate the Safety and Exploratory Efficacy of GC1119

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