Evaluate the Safety and Exploratory Efficacy of GC1119

NCT01653444 | Completed Phase 1

• 1 other identifier: GC1119_P1
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 3

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.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events

  10weeks

Secondary Outcomes (8)

• 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

Study Arms (2)

GC1119 0.5 mg/kg

EXPERIMENTAL

0.5 mg/kg biweekly

Drug: GC1119

GC1119 1.0 mg/kg

EXPERIMENTAL

1.0 mg/kg biweekly

Drug: GC1119

Interventions

GC1119 DRUG

biweekly, IV infusion

GC1119 0.5 mg/kg; GC1119 1.0 mg/kg

Eligibility Criteria

• Age: 16 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Green Cross Corporation: lead

Study Sites (3)

Asan Medical Center

Songpa-gu, Seoul, South Korea

Location

Soon Cung Hyang University Hospital

Yongsan-gu, Seoul, South Korea

Location

Ajou University School of Medicine

Yeongtong-gu, Suwon, South Korea

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2012
• First Posted: July 31, 2012
• Study Start: November 1, 2012
• Primary Completion: May 1, 2014
• Study Completion: October 1, 2015
• Last Updated: June 24, 2016

Record last verified: 2016-06

Data Sharing

• IPD Sharing: Will not share

Locations

KR (3)