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

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.

Study Overview

• Status: Unknown
• Conditions: Mucopolysaccharidosis II
• Intervention / Treatment: Biological: idursulfase beta; Biological: idursulfase

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.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 35 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm 1; 0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks	Biological: idursulfase beta; IV, weekly infusion for 24 weeks; Other Names: GC1111
Experimental: Arm 2; 1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks	Biological: idursulfase beta; IV, weekly infusion for 24 weeks; Other Names: GC1111
Experimental: Arm 3; 1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks	Biological: idursulfase beta; IV, weekly infusion for 24 weeks; Other Names: GC1111
Active Comparator: Arm 4; 0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks	Biological: idursulfase; 0.5 mg/kg, iv, weekly infusion for 24 weeks; Other Names: Elaprase

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25	Baseline to Week 25

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Green Cross Corporation

Study record dates

Study Major Dates

• Study Start: December 1, 2016
• Primary Completion (Anticipated): December 1, 2019
• Study Completion (Anticipated): June 1, 2020

Study Registration Dates

• First Submitted: January 17, 2016
• First Submitted That Met QC Criteria: January 21, 2016
• First Posted (Estimate): January 26, 2016

Study Record Updates

• Last Update Posted (Estimate): January 26, 2016
• Last Update Submitted That Met QC Criteria: January 21, 2016
• Last Verified: January 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidosis II; Mucopolysaccharidoses
• Other Study ID Numbers: GC1111B_P2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No