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Safety and Efficacy of Hunterase (GC1111)

Primary Purpose

Hunter Syndrome

Status
Completed
Phase
Phase 3
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Hunterase
Sponsored by
Green Cross Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hunter Syndrome focused on measuring Hunter syndrome, idursulfase-beta, Hunterase, GC1111

Eligibility Criteria

undefined - 5 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  1. The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:

    • as measured in plasma, leukocytes, or fibroblasts,

      • a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the lower limit of the normal range

    • That corresponds to one or more of the following:

      • a normal enzyme activity level of one other sulfatase
      • Confirmed as MPS2 by genetic test results
      • shows clinical symptoms/ visible signs of MPS2
  2. < 6 years old and male
  3. Patients who are able to comply with the study requirements
  4. The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study

Exclusion Criteria:

  1. The patient has had a tracheostomy
  2. The patient has known severe hypersensitivity or shock to any of the components of idursulfase
  3. The patient has received treatment with another investigational therapy within 30 days prior to enrollment
  4. History of a stem cell transplant
  5. The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)

Sites / Locations

  • Samsug Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Test drug

Arm Description

Idursulfase-beta

Outcomes

Primary Outcome Measures

Incidence of adverse events

Secondary Outcome Measures

change of anti-idursulfase-beta antibody status
Percent Change of Urine GAG

Full Information

First Posted
July 16, 2012
Last Updated
July 7, 2014
Sponsor
Green Cross Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT01645189
Brief Title
Safety and Efficacy of Hunterase
Acronym
GC1111
Official Title
To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy
Study Type
Interventional

2. Study Status

Record Verification Date
July 2014
Overall Recruitment Status
Completed
Study Start Date
July 2012 (undefined)
Primary Completion Date
September 2013 (Actual)
Study Completion Date
September 2013 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hunter Syndrome
Keywords
Hunter syndrome, idursulfase-beta, Hunterase, GC1111

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Test drug
Arm Type
Experimental
Arm Description
Idursulfase-beta
Intervention Type
Biological
Intervention Name(s)
Hunterase
Intervention Description
once weekly, 0.5mg/kg IV infusion
Primary Outcome Measure Information:
Title
Incidence of adverse events
Time Frame
One year
Secondary Outcome Measure Information:
Title
change of anti-idursulfase-beta antibody status
Time Frame
baseline and one year
Title
Percent Change of Urine GAG
Time Frame
baseline to 53 weeks

10. Eligibility

Sex
Male
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: The patient has a diagnosis of Hunter syndrome based upon biochemical criteria: as measured in plasma, leukocytes, or fibroblasts, a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the lower limit of the normal range That corresponds to one or more of the following: a normal enzyme activity level of one other sulfatase Confirmed as MPS2 by genetic test results shows clinical symptoms/ visible signs of MPS2 < 6 years old and male Patients who are able to comply with the study requirements The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study Exclusion Criteria: The patient has had a tracheostomy The patient has known severe hypersensitivity or shock to any of the components of idursulfase The patient has received treatment with another investigational therapy within 30 days prior to enrollment History of a stem cell transplant The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dong-Kyu Jin
Organizational Affiliation
Samsung medical center, Seoul, Republic of Korea
Official's Role
Principal Investigator
Facility Information:
Facility Name
Samsug Medical Center
City
Seoul
Country
Korea, Republic of

12. IPD Sharing Statement

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Safety and Efficacy of Hunterase

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