NCT02663024

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

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

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Green Cross Corporation · Industry
• Sex: Male
• Age: 5 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Mucopolysaccharidosis II

Interventions

Type	Name	Description
BIOLOGICAL	idursulfase beta	IV, weekly infusion for 24 weeks
BIOLOGICAL	idursulfase	0.5 mg/kg, iv, weekly infusion for 24 weeks

Timeline

• Start date: 2016-12-01
• Primary completion: 2019-12-01
• Completion: 2020-06-01
• First posted: 2016-01-26
• Last updated: 2016-01-26