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Completed

NCT Number: NCT01653444

Evaluate the Safety and Exploratory Efficacy of GC1119

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.

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

GC1119

Drug

biweekly, IV infusion

Primary outcomes

  1. Incidence of adverse events

    Time frame: 10weeks

Secondary outcomes

  1. change and %change of Plasma GL-3 concentration

    Time frame: baseline and 10weeks

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

    Time frame: 10weeks

  3. change and %change of urine GL-3 concentration

    Time frame: baseline and 10weeks

  4. change and %change of kidney function

    Time frame: baseline and 10weeks

  5. change and %change of kidney size

    Time frame: baseline and 10weeks

  6. change and %change of heart size

    Time frame: baseline and 10weeks

  7. change of results of cornial opacity examination

    Time frame: baseline and 10weeks

  8. change of scores that are measured by pain questionnaire

    Time frame: baseline and 10weeks

Sponsors and collaborators

Lead sponsor

Green Cross Corporation

Industry

Registry information

Official study 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

Important dates

Study start
2012
Primary completion
2014
Study completion
2015
First posted
Jul 31, 2012
Registry last updated
Jun 24, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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