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To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

Primary Purpose

Mucopolysaccharidosis II

Status
Completed
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
GC1111_0.5mg/kg
GC1111_1.0mg/kg
Elaprase_0.5mg/kg
Sponsored by
Green Cross Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis II focused on measuring Hunter syndrome, MPS II, Idursulfase

Eligibility Criteria

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

Inclusion Criteria:

  1. Patients with diagnosis of MPS II based on both clinical and biochemical criteria
  2. Male, ages 6 to 35 years old
  3. Patients who are able to comply with the study requirements
  4. Patients who have given voluntary written consent to participate in the study
  5. Patients who is acceptable for using an appropriate method of contraception

Exclusion Criteria:

  1. History of a tracheostomy or a bone marrow transplant
  2. Known hypersensitivity to idursulfase
  3. Known shock to idursulfase
  4. History of receiving treatment with another investigational therapy within the past 30 days
  5. History of a stem cell transplant
  6. Known hypersensitivity to any of the components of idursulfase
  7. Female

Sites / Locations

  • Samsung Medical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Active Comparator

Arm Label

GC1111_0.5mg/kg

GC1111_1.0mg/kg

Elaprase_0.5mg/kg

Arm Description

Outcomes

Primary Outcome Measures

Primary Outcome
measurement of % change of Urine GAG

Secondary Outcome Measures

Secondary Outcome
Measurements of the six-minute-walk test (6-MWT) Measurements of liver volume Measurements of heart size and heart function Measurements of joint range of motion Measurements of urine GAG levels Measurements of pulmonary function Evaluate the safety: vital signs, physical examination, laboratory tests, adverse events, immunogenecity

Full Information

First Posted
February 22, 2011
Last Updated
February 12, 2012
Sponsor
Green Cross Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT01301898
Brief Title
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
Official Title
Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
Study Type
Interventional

2. Study Status

Record Verification Date
February 2012
Overall Recruitment Status
Completed
Study Start Date
May 2010 (undefined)
Primary Completion Date
March 2011 (Actual)
Study Completion Date
March 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Green Cross Corporation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis II
Keywords
Hunter syndrome, MPS II, Idursulfase

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
31 (Actual)

8. Arms, Groups, and Interventions

Arm Title
GC1111_0.5mg/kg
Arm Type
Experimental
Arm Title
GC1111_1.0mg/kg
Arm Type
Experimental
Arm Title
Elaprase_0.5mg/kg
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
GC1111_0.5mg/kg
Intervention Type
Drug
Intervention Name(s)
GC1111_1.0mg/kg
Intervention Type
Drug
Intervention Name(s)
Elaprase_0.5mg/kg
Primary Outcome Measure Information:
Title
Primary Outcome
Description
measurement of % change of Urine GAG
Time Frame
baseline, every 4 weeks
Secondary Outcome Measure Information:
Title
Secondary Outcome
Description
Measurements of the six-minute-walk test (6-MWT) Measurements of liver volume Measurements of heart size and heart function Measurements of joint range of motion Measurements of urine GAG levels Measurements of pulmonary function Evaluate the safety: vital signs, physical examination, laboratory tests, adverse events, immunogenecity
Time Frame
baseline, every 12 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with diagnosis of MPS II based on both clinical and biochemical criteria Male, ages 6 to 35 years old Patients who are able to comply with the study requirements Patients who have given voluntary written consent to participate in the study Patients who is acceptable for using an appropriate method of contraception Exclusion Criteria: History of a tracheostomy or a bone marrow transplant Known hypersensitivity to idursulfase Known shock to idursulfase History of receiving treatment with another investigational therapy within the past 30 days History of a stem cell transplant Known hypersensitivity to any of the components of idursulfase Female
Facility Information:
Facility Name
Samsung Medical Center
City
Seoul
State/Province
Irwon-dong, Gangnam-Gu
ZIP/Postal Code
135-710
Country
Korea, Republic of

12. IPD Sharing Statement

Citations:
PubMed Identifier
23497636
Citation
Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, Han SJ, Jin DK. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome). Orphanet J Rare Dis. 2013 Mar 18;8:42. doi: 10.1186/1750-1172-8-42.
Results Reference
derived

Learn more about this trial

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

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