Study of Idursulfase-beta (GC1111) in Hunter Syndrome
- Conditions
- Mucopolysaccharidosis II
- Registration Number
- NCT02663024
- Lead Sponsor
- Green Cross Corporation
- 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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- Male
- Target Recruitment
- 20
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25 Baseline to Week 25
- Secondary Outcome Measures
Name Time Method Safety changes from baseline in clinical laboratory tests, physical examination and vital signs Baseline to Week 25 Immunogenicity Baseline to Week 25 anti-drug-antibody
Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax) 1 and 17 week Change from baseline in Six Minute Walk Test at Week 25 Baseline to Week 25 Incidence of Adverse Events and Serious Adverse Events Baseline to Week 25 Percent change from baseline in Six Minute Walk Test at Week 25 Baseline to Week 25 Change from baseline in Liver volume at Week 25 Baseline to Week 25 Liver volume measured by MRI
Change from baseline in Spleen volume at Week 25 Baseline to Week 25 Spleen volume measured by MRI
Pharmacokinetic profile - Area under the serum concentration time curve (AUClast) 1 and 17 week Change from baseline in urinary GAG at Week 25 Baseline to Week 25 Percent change from baseline in Liver volume at Week 25 Baseline to Week 25 Liver volume measured by MRI
Percent change from baseline in Spleen volume at Week 25 Baseline to Week 25 Spleen volume measured by MRI
Pharmacokinetic profile - Time at which Cmax is observed (Tmax) 1 and 17 week