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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

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

    Background - CRIS Registration Number, Unique Protocol ID, Public/Brief Title, Scientific Title, Acronym, MFDS Regulated Study, IND/IDE Protocol, Registered at Other Registry, Name of Registry/Registration Number
    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

    Institutional Review Board Information
    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 Details Information - Contact Person for Principal Investigator / Scientific Queries, Contact Person for Public Queries, Contact Person for Updating Information의 Name, Title, Email, Telephone, Cellular Phone, Affiliation, Address
    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

    Status Information - Study Site, Overall Recruitment Status, Date of First Enrollment, Status of First Enrollment, Target Number of Participant, Primary Completion Date, Recruitment Status by Participating Study Site, Name of Study Site, Recruitment Status, Date of First Enrollment, Status of First Enrollemnt
    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

    Source of Monetary / Material Support Information - Organization Name, Organization Type, Project ID
    1. Source of Monetary/Material Support
    Organization Name Green Cross
    Organization Type Pharmaceutical Company
    Project ID GC1119
  • 6. Sponsor Organization

    Sponsor Organization Information - Organization Name, Organization Type
    1. Sponsor Organization
    Organization Name Green Cross
    Organization Type Pharmaceutical Company
  • 7. Study Summary

    Study Summary Information
    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 Design Information - Study Type, Study Purpose, Phase, Intervention Model, Blinding/Masking, Blinded Subject, Allocation, Intervention Type, Intervention Description, Number of Arms, Arm Label, Target Number of Participant, Arm Type, Arm Description
    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

    Subject Eligibility Information
    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)

    Outcome Measure(s) Information - Type of Primary Outcome, Primary Outcome, Outcome, Timepoint, Secondary Outcome, Outcome, Timepoint
    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

    Study Results and Publication Information - Result Registered, Final Enrollment Number, Number of Publication, Publications, Results Upload, Date of Posting Results, Protocol URL or File Upload, Brief Summary
    Result Registered
  • 12. Sharing of Study Data(Deidentified Individual-Patient Data, IPD)

    Sharing of Study Data Information - Sharing Statement, Time of Sharing, Way of Sharing
    Sharing Statement Not provided at time of Registration
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