Study of Idursulfase-beta (GC1111) in Hunter Syndrome
Primary Purpose
Mucopolysaccharidosis II
Status
Unknown status
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
idursulfase beta
idursulfase
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis II
Eligibility Criteria
Inclusion Criteria:
- Male patients between 5 and 35 years of age
- Informed consent form signed
- Patients diagnosed with hunter syndrome
- Previously untreated with an enzyme replacement therapy
Exclusion Criteria:
- History of tracheostomy, bone marrow transplant, or cord blood transplant
- Treatment with another investigational product within 30 days prior to the start of study drug
- Known hypersensitivity of any of the ingredients of study drug
- Patient with severe hunter syndrome who cannot perform 6MWT
- Female patients
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Active Comparator
Arm Label
Arm 1
Arm 2
Arm 3
Arm 4
Arm Description
0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks
Outcomes
Primary Outcome Measures
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
Secondary Outcome Measures
Change from baseline in urinary GAG at Week 25
Change from baseline in Six Minute Walk Test at Week 25
Percent change from baseline in Six Minute Walk Test at Week 25
Change from baseline in Liver volume at Week 25
Liver volume measured by MRI
Percent change from baseline in Liver volume at Week 25
Liver volume measured by MRI
Change from baseline in Spleen volume at Week 25
Spleen volume measured by MRI
Percent change from baseline in Spleen volume at Week 25
Spleen volume measured by MRI
Incidence of Adverse Events and Serious Adverse Events
Safety changes from baseline in clinical laboratory tests, physical examination and vital signs
Immunogenicity
anti-drug-antibody
Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)
Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)
Pharmacokinetic profile - Time at which Cmax is observed (Tmax)
Full Information
NCT ID
NCT02663024
First Posted
January 17, 2016
Last Updated
January 21, 2016
Sponsor
Green Cross Corporation
1. Study Identification
Unique Protocol Identification Number
NCT02663024
Brief Title
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
Official Title
Phase 2, Randomized, Double-blind, Active-controlled, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Idursulfase-beta (GC1111) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
Study Type
Interventional
2. Study Status
Record Verification Date
January 2016
Overall Recruitment Status
Unknown status
Study Start Date
December 2016 (undefined)
Primary Completion Date
December 2019 (Anticipated)
Study Completion Date
June 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Green Cross Corporation
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study evaluates the efficacy and safety of three doses of GC1111 in patients with Hunter Syndrome. Participants will be randomized to one of three doses of GC1111 or comparator.
Detailed Description
This is a randomized, double-blind, active-controlled, dose-ranging study, where patient will receive one of the three doses of GC1111 (0.5 mg/kg, 1.0 mg/kg, and 1.5 mg/kg) or ELAPRASE 0.5 mg/kg. Approximately 20 patients will be administrated each study drug once every week as an iv infusion for 24 weeks. Efficacy of GC1111 will be evaluated in Six-Minute Walk Test (6MWT), urine Glycosaminoglycans(uGAG), liver and spleen volume, percent predicted Forced Vital Capacity(FVC), and cardiac size and function. Also immunogenicity, Pharmacokinetics(PK) and safety will be evaluated.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis II
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Arm 1
Arm Type
Experimental
Arm Description
0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
Arm Title
Arm 2
Arm Type
Experimental
Arm Description
1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
Arm Title
Arm 3
Arm Type
Experimental
Arm Description
1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
Arm Title
Arm 4
Arm Type
Active Comparator
Arm Description
0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks
Intervention Type
Biological
Intervention Name(s)
idursulfase beta
Other Intervention Name(s)
GC1111
Intervention Description
IV, weekly infusion for 24 weeks
Intervention Type
Biological
Intervention Name(s)
idursulfase
Other Intervention Name(s)
Elaprase
Intervention Description
0.5 mg/kg, iv, weekly infusion for 24 weeks
Primary Outcome Measure Information:
Title
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
Time Frame
Baseline to Week 25
Secondary Outcome Measure Information:
Title
Change from baseline in urinary GAG at Week 25
Time Frame
Baseline to Week 25
Title
Change from baseline in Six Minute Walk Test at Week 25
Time Frame
Baseline to Week 25
Title
Percent change from baseline in Six Minute Walk Test at Week 25
Time Frame
Baseline to Week 25
Title
Change from baseline in Liver volume at Week 25
Description
Liver volume measured by MRI
Time Frame
Baseline to Week 25
Title
Percent change from baseline in Liver volume at Week 25
Description
Liver volume measured by MRI
Time Frame
Baseline to Week 25
Title
Change from baseline in Spleen volume at Week 25
Description
Spleen volume measured by MRI
Time Frame
Baseline to Week 25
Title
Percent change from baseline in Spleen volume at Week 25
Description
Spleen volume measured by MRI
Time Frame
Baseline to Week 25
Title
Incidence of Adverse Events and Serious Adverse Events
Time Frame
Baseline to Week 25
Title
Safety changes from baseline in clinical laboratory tests, physical examination and vital signs
Time Frame
Baseline to Week 25
Title
Immunogenicity
Description
anti-drug-antibody
Time Frame
Baseline to Week 25
Title
Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)
Time Frame
1 and 17 week
Title
Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)
Time Frame
1 and 17 week
Title
Pharmacokinetic profile - Time at which Cmax is observed (Tmax)
Time Frame
1 and 17 week
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male patients between 5 and 35 years of age
Informed consent form signed
Patients diagnosed with hunter syndrome
Previously untreated with an enzyme replacement therapy
Exclusion Criteria:
History of tracheostomy, bone marrow transplant, or cord blood transplant
Treatment with another investigational product within 30 days prior to the start of study drug
Known hypersensitivity of any of the ingredients of study drug
Patient with severe hunter syndrome who cannot perform 6MWT
Female patients
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sangmi Kang
Phone
82-31-260-1957
Email
sm6220@greencross.com
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
We'll reach out to this number within 24 hrs