Clinical trial NCT01653444: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Fabry Disease
Lead sponsor: GC Biopharma Corp
Sponsor class: INDUSTRY
First posted: 2012-07-31
Enrollment: 8
Sex: MALE
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Incidence of adverse events
timeFrame: 10weeks
Secondary outcomes:
measure: change and %change of Plasma GL-3 concentration
timeFrame: baseline and 10weeks
measure: The ratio of subjects whose plasma GL-3 values are within reference range
timeFrame: 10weeks
measure: change and %change of urine GL-3 concentration
timeFrame: baseline and 10weeks
measure: change and %change of kidney function
timeFrame: baseline and 10weeks
measure: change and %change of kidney size
timeFrame: baseline and 10weeks
measure: change and %change of heart size
timeFrame: baseline and 10weeks
measure: change of results of cornial opacity examination
timeFrame: baseline and 10weeks
measure: change of scores that are measured by pain questionnaire
timeFrame: baseline and 10weeks
Other outcomes:
Locations:
city: Songpa-gu
country: Korea, Republic of
facility: Asan Medical Center
state: Seoul
city: Yongsan-gu
country: Korea, Republic of
facility: Soon Cung Hyang University Hospital
state: Seoul
city: Yeongtong-gu
country: Korea, Republic of
facility: Ajou University School of Medicine
state: Suwon
Study officials:
References: