Status Approved
First Submitted Date
2012/07/26
Registered Date
2012/12/03
Last Updated Date
2012/11/30
CRIS Required
WHO ICTRP (International Clinical Trial Registry Platform) Required
1. Background
CRIS Registration Number |
KCT0000597 |
---|---|
Unique Protocol ID | 2012-0351 |
Public/Brief 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 |
Scientific 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 |
Acronym | |
MFDS Regulated Study | Yes |
IND/IDE Protocol | Yes |
Registered at Other Registry | Yes |
Name of Registry / Registration Number | ClinicalTrials.gov-NCT01653444 |
Healthcare Benefit Approval Status |
2. Institutional Review Board / Ethics Committee
Board Approval Status | Submitted approval |
---|---|
Board Approval Number | 2012-0351 |
Approval Date | 2012-06-15 |
Institutional Review Board Name | Asan Medical Center Institutional Review Board |
Institutional Review Board Address | |
Institutional Review Board Telephone | |
Data Monitoring Committee |
3. Contact Details
Contact Person for Principal Investigator / Scientific Queries | |
---|---|
Name | Hanwook Yoo |
Title | MD |
Telephone | +82-2-3010-3374 |
Affiliation | Asan Medical Center |
Address | |
Contact Person for Public Queries | |
Name | Ji Hye Song |
Title | CRC |
Telephone | +82-2-3010-8495 |
Affiliation | Asan Medical Center |
Address | |
Contact Person for Updating Information | |
Name | Ahmi Woo |
Title | Assistant Manager |
Telephone | +82-31-260-9467 |
Affiliation | Green Cross |
Address |
4. Status
Study Site | Multi-center Number of center : 3 | |
---|---|---|
Overall Recruitment Status | Recruiting | |
Date of First Enrollment | 2012-11-14 Actual | |
Target Number of Participant | 10 | |
Primary Completion Date | ||
Study Completion Date | ||
Recruitment Status by Participating Study Site 1 | ||
Name of Study | Asan Medical Center | |
Recruitment Status | Recruiting | |
Date of First Enrollment | 2012-11-14 , | |
Recruitment Status by Participating Study Site 2 | ||
Name of Study | Ajou University Hospital | |
Recruitment Status | Not yet recruiting | |
Date of First Enrollment | 2012-12-01 , | |
Recruitment Status by Participating Study Site 3 | ||
Name of Study | Soon Chun Hyang University Hospital Seoul | |
Recruitment Status | Not yet recruiting | |
Date of First Enrollment | 2012-12-01 , |
5. Source of Monetary / Material Support
1. Source of Monetary/Material Support | |
---|---|
Organization Name | Green Cross |
Organization Type | Pharmaceutical Company |
Project ID | GC1119 |
6. Sponsor Organization
1. Sponsor Organization | |
---|---|
Organization Name | Green Cross |
Organization Type | Pharmaceutical Company |
7. Study Summary
Lay Summary | <Purpose> 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. <Subjects> Fabry patients <Procedure> 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. Five patients on step 1 will recieve 0.5 mg/kg via intravenous infusion of GC1119 every 2 weeks for 10 weeks (total 5 infusions), and will be evaluated the safety and exploratory efficacy. After treatment of GC1119, acute adverse event will be monitored for 3 hours. Subject enrolment to step 2 will be started when anaphylaxix, severe allergic reaction or severe infusion reaction is showed under one subjects (under 20%) in step 1 and when at least one subjects is treated study drug five times and safety is confirmed. When safety is confirmed in step 1, 5 patients on step 2 will recive 1.0 mg/kg via intravenous infusion every 2 weeks for 10 weeks (total 5 infusions), and will be evaluated the safety and exploratory efficacy. |
---|
8. Study Design
Study Type | Interventional Study |
---|---|
Study Purpose | Treatment |
Phase | Phase1 |
Intervention Model | Single Group |
Blinding/Masking | Open |
Allocation | Not Applicable |
Intervention Type | Drug |
Intervention Description | Five patients on step 1 will recieve 0.5 mg/kg via intravenous infusion of GC1119 every 2 weeks for 10 weeks (total 5 infusions), and will be evaluated the safety and exploratory efficacy. Patients will be evaluated acute infusion reaction, such as anaphylaxis, severe allergic reaction or severe infusion related reaction after the infusion for 3 hours. If the incidence of infusion reaction is less than one (less than 20%) patient of 5 patiens on step 1 who recieve 2 infusions, and safety is confirmed in at least one patines who recieve all 5 treatments, subjects can be enrolled on step 2 (1.0 mg/kg). When safety is confirmed in step 1, 5 patients on step 2 will recive 1.0 mg/kg via intravenous infusion every 2 weeks for 10 weeks (total 5 infusions), and will be evaluated the safety and exploratory efficacy. |
Number of Arms | 1 |
Arm 1 |
Arm Label GC1119 |
Target Number of Participant 10 |
|
Arm Type Experimental |
|
Arm Description Five patients on step 1 will recieve 0.5 mg/kg via intravenous infusion of GC1119 every 2 weeks for 10 weeks (total 5 infusions), and will be evaluated the safety and exploratory efficacy. When safety is confirmed in step 1, 5 patients on step 2 will recive 1.0 mg/kg via intravenous infusion every 2 weeks for 10 weeks (total 5 infusions), and will be evaluated the safety and exploratory efficacy. |
9. Subject Eligibility
Condition(s)/Problem(s) |
* (E00-E90)Endocrine, nutritional and metabolic diseases (E75.2)Other sphingolipidosis |
---|---|
Rare Disease | No |
Inclusion Criteria |
Gender Male |
Age 16Year~No Limit |
|
Description 1) Subjects with a current diagnosis of Fabry's disease (leukocyte α-gal activity of ≤ 10 mnol/hr/ml and a mutation in α-galactosidase A gene) 2) Males ≥ 16 years old 3) Subjects capable of performing this clinical trial in an appropriate manner 4) 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 5) Agreement to contraception during the study period |
|
Exclusion Criteria |
1) Serum creatinine > 2.5mg/dl 2) Subjects have a plan to kidney transplantation 3) Subjects have undergone kidney transplantation 4) Subjects are currently on dialysis 5) 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 6) Known life-threatening hypersensitivity(anaphylactic reaction) to α-galactosidase 7) 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 8) Known hypersensitivity to any of the ingredients of study drug(including excipients) 9) Subjects need the medication of prohibited drug 10) Alcoholism or drug addiction |
Healthy Volunteers | No |
10. Outcome Measure(s)
Type of Primary Outcome | Safety | |
---|---|---|
Primary Outcome(s) 1 | ||
Outcome | Incidence of adverse events |
|
Timepoint | 10 weeks |
|
Secondary Outcome(s) 1 | ||
Outcome | Change and % change of Plasma GL-3 concentration |
|
Timepoint | Baseline and 10 weeks |
|
Secondary Outcome(s) 2 | ||
Outcome | The ratio of subjects whose plasma GL-3 values are within reference range |
|
Timepoint | 10 weeks |
|
Secondary Outcome(s) 3 | ||
Outcome | Change and % change of urine GL-3 concentration |
|
Timepoint | Baseline and 10 weeks |
|
Secondary Outcome(s) 4 | ||
Outcome | Change and % change of kidney function |
|
Timepoint | Baseline and 10 weeks |
|
Secondary Outcome(s) 5 | ||
Outcome | Change and % change of heart size |
|
Timepoint | Baseline and 10 weeks |
|
Secondary Outcome(s) 6 | ||
Outcome | Change of scores that are measured by pain questionnaire |
|
Timepoint | Baseline and 10 weeks |
11. Study Results and Publication
Result Registered |
---|
12. Sharing of Study Data(Deidentified Individual-Patient Data, IPD)
Sharing Statement | Not provided at time of Registration |
---|
TOP
BOTTOM
화면 최하단으로 이동