Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Mucopolysaccharidosis II Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02663024
• Other study ID #: GC1111B_P2
• Status: Not yet recruiting
• Phase: Phase 2
• First received: January 17, 2016
• Last updated: January 21, 2016
• Start date: December 2016
• Est. completion date: June 2020

Study information

• Verified date: January 2016
• Source: Green Cross Corporation
• Contact: Sangmi Kang
• Phone: 82-31-260-1957
• Email: sm6220[@]greencross.com
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: June 2020
• Est. primary completion date: December 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 35 Years

Eligibility

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 Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis II

Intervention

Biological:
• idursulfase beta
IV, weekly infusion for 24 weeks
• idursulfase
0.5 mg/kg, iv, weekly infusion for 24 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Green Cross Corporation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25		Baseline to Week 25	No
Secondary	Change from baseline in urinary GAG at Week 25		Baseline to Week 25	No
Secondary	Change from baseline in Six Minute Walk Test at Week 25		Baseline to Week 25	No
Secondary	Percent change from baseline in Six Minute Walk Test at Week 25		Baseline to Week 25	No
Secondary	Change from baseline in Liver volume at Week 25	Liver volume measured by MRI	Baseline to Week 25	No
Secondary	Percent change from baseline in Liver volume at Week 25	Liver volume measured by MRI	Baseline to Week 25	No
Secondary	Change from baseline in Spleen volume at Week 25	Spleen volume measured by MRI	Baseline to Week 25	No
Secondary	Percent change from baseline in Spleen volume at Week 25	Spleen volume measured by MRI	Baseline to Week 25	No
Secondary	Incidence of Adverse Events and Serious Adverse Events		Baseline to Week 25	Yes
Secondary	Safety changes from baseline in clinical laboratory tests, physical examination and vital signs		Baseline to Week 25	Yes
Secondary	Immunogenicity	anti-drug-antibody	Baseline to Week 25	Yes
Secondary	Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)		1 and 17 week	No
Secondary	Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)		1 and 17 week	No
Secondary	Pharmacokinetic profile - Time at which Cmax is observed (Tmax)		1 and 17 week	No