Clinical Trial: Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

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 Synd

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 Summary: 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.
Sponsor: Green Cross Corporation

Current Primary Outcome: Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25 [ Time Frame: Baseline to Week 25 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change from baseline in urinary GAG at Week 25 [ Time Frame: Baseline to Week 25 ]
  • Change from baseline in Six Minute Walk Test at Week 25 [ Time Frame: Baseline to Week 25 ]
  • Percent change from baseline in Six Minute Walk Test at Week 25 [ Time Frame: Baseline to Week 25 ]
  • Change from baseline in Liver volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Liver volume measured by MRI
  • Percent change from baseline in Liver volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Liver volume measured by MRI
  • Change from baseline in Spleen volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Spleen volume measured by MRI
  • Percent change from baseline in Spleen volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Spleen volume measured by MRI
  • Incidence of Adverse Events and Serious Adverse Events [ Time Frame: Baseline to Week 25 ]
  • Safety changes from baseline in clinical laboratory tests, physical examination and vital signs [ Time Frame: Baseline to Week 25 ]
  • Immunogenicity [ Time Frame: Baseline to Week 25 ]
    anti-drug-antibody
  • Pharmacokinetic profile - Area under the serum concentration time curve (AUClast) [ Time Frame: 1 and 17 week ]
  • Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax) [ Time Frame: 1 and 17 week ]
  • Pharmacokinetic profile - Time at which Cmax is observed (Tmax) [ Time Frame: 1 and 17 week ]


Original Secondary Outcome: Same as current

Information By: Green Cross Corporation

Dates:
Date Received: January 17, 2016
Date Started: December 2016
Date Completion: June 2020
Last Updated: January 21, 2016
Last Verified: January 2016