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Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Primary Purpose

Mucopolysaccharidosis II

Status

Unknown status

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

idursulfase beta

idursulfase

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis II

Eligibility Criteria

5 Years - 35 Years (Child, Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Active Comparator

Arm Label

Arm 1

Arm 2

Arm 3

Arm 4

Arm Description

0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks

Outcomes

Primary Outcome Measures

Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25

Secondary Outcome Measures

Change from baseline in urinary GAG at Week 25

Change from baseline in Six Minute Walk Test at Week 25

Percent change from baseline in Six Minute Walk Test at Week 25

Change from baseline in Liver volume at Week 25

Liver volume measured by MRI

Percent change from baseline in Liver volume at Week 25

Liver volume measured by MRI

Change from baseline in Spleen volume at Week 25

Spleen volume measured by MRI

Percent change from baseline in Spleen volume at Week 25

Spleen volume measured by MRI

Incidence of Adverse Events and Serious Adverse Events

Safety changes from baseline in clinical laboratory tests, physical examination and vital signs

Immunogenicity

anti-drug-antibody

Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)

Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)

Pharmacokinetic profile - Time at which Cmax is observed (Tmax)

Full Information

NCT ID

NCT02663024

First Posted

January 17, 2016

Last Updated

January 21, 2016

Sponsor

Green Cross Corporation

1. Study Identification

Unique Protocol Identification Number

NCT02663024

Brief Title

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2016

Overall Recruitment Status

Unknown status

Study Start Date

December 2016 (undefined)

Primary Completion Date

December 2019 (Anticipated)

Study Completion Date

June 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Green Cross Corporation

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Mucopolysaccharidosis II

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Arm 1

Arm Type

Experimental

Arm Description

0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Arm Title

Arm 2

Arm Type

Experimental

Arm Description

1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Arm Title

Arm 3

Arm Type

Experimental

Arm Description

1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Arm Title

Arm 4

Arm Type

Active Comparator

Arm Description

0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks

Intervention Type

Biological

Intervention Name(s)

idursulfase beta

Other Intervention Name(s)

GC1111

Intervention Description

IV, weekly infusion for 24 weeks

Intervention Type

Biological

Intervention Name(s)

idursulfase

Other Intervention Name(s)

Elaprase

Intervention Description

0.5 mg/kg, iv, weekly infusion for 24 weeks

Primary Outcome Measure Information:

Title

Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25

Time Frame

Baseline to Week 25

Secondary Outcome Measure Information:

Title

Change from baseline in urinary GAG at Week 25

Time Frame

Baseline to Week 25

Title

Change from baseline in Six Minute Walk Test at Week 25

Time Frame

Baseline to Week 25

Title

Percent change from baseline in Six Minute Walk Test at Week 25

Time Frame

Baseline to Week 25

Title

Change from baseline in Liver volume at Week 25

Description

Liver volume measured by MRI

Time Frame

Baseline to Week 25

Title

Percent change from baseline in Liver volume at Week 25

Description

Liver volume measured by MRI

Time Frame

Baseline to Week 25

Title

Change from baseline in Spleen volume at Week 25

Description

Spleen volume measured by MRI

Time Frame

Baseline to Week 25

Title

Percent change from baseline in Spleen volume at Week 25

Description

Spleen volume measured by MRI

Time Frame

Baseline to Week 25

Title

Incidence of Adverse Events and Serious Adverse Events

Time Frame

Baseline to Week 25

Title

Safety changes from baseline in clinical laboratory tests, physical examination and vital signs

Time Frame

Baseline to Week 25

Title

Immunogenicity

Description

anti-drug-antibody

Time Frame

Baseline to Week 25

Title

Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)

Time Frame

1 and 17 week

Title

Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)

Time Frame

1 and 17 week

Title

Pharmacokinetic profile - Time at which Cmax is observed (Tmax)

Time Frame

1 and 17 week

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Sangmi Kang

Phone

82-31-260-1957

sm6220@greencross.com

12. IPD Sharing Statement

Plan to Share IPD

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Study of Idursulfase-beta (GC1111) in Hunter Syndrome

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