Evaluate the Safety and Exploratory Efficacy of GC1119

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01653444
• Other study ID #: GC1119_P1
• Status: Completed
• Phase: Phase 1
• First received: July 24, 2012
• Last updated: June 23, 2016
• Start date: November 2012
• Est. completion date: October 2015

Study information

• Verified date: June 2016
• Source: Green Cross Corporation
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: October 2015
• Est. primary completion date: May 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 16 Years and older

Eligibility

Inclusion Criteria:

• Subjects with a current diagnosis of Fabry's disease

• Plasma a-gal activity of = 1.5mnol/hr/ml and have a mutation in a-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 a-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 Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• GC1119
biweekly, IV infusion

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Green Cross Corporation

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events		10weeks	Yes
Secondary	change and %change of Plasma GL-3 concentration		baseline and 10weeks	No
Secondary	The ratio of subjects whose plasma GL-3 values are within reference range		10weeks	No
Secondary	change and %change of urine GL-3 concentration		baseline and 10weeks	No
Secondary	change and %change of kidney function		baseline and 10weeks	No
Secondary	change and %change of kidney size		baseline and 10weeks	No
Secondary	change and %change of heart size		baseline and 10weeks	No
Secondary	change of results of cornial opacity examination		baseline and 10weeks	No
Secondary	change of scores that are measured by pain questionnaire		baseline and 10weeks	No