Safety and Efficacy of Hunterase (GC1111)

This study has been completed.
Information provided by (Responsible Party):
Green Cross Corporation Identifier:
First received: July 16, 2012
Last updated: July 7, 2014
Last verified: July 2014
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.

Condition Intervention Phase
Hunter Syndrome
Biological: Hunterase
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Green Cross Corporation:

Primary Outcome Measures:
  • Incidence of adverse events [ Time Frame: One year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • change of anti-idursulfase-beta antibody status [ Time Frame: baseline and one year ] [ Designated as safety issue: Yes ]
  • Percent Change of Urine GAG [ Time Frame: baseline to 53 weeks ] [ Designated as safety issue: No ]

Enrollment: 6
Study Start Date: July 2012
Study Completion Date: September 2013
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Test drug
Biological: Hunterase
once weekly, 0.5mg/kg IV infusion


Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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)
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Please refer to this study by its identifier: NCT01645189

Korea, Republic of
Samsug Medical Center
Seoul, Korea, Republic of
Sponsors and Collaborators
Green Cross Corporation
Principal Investigator: Dong-Kyu Jin Samsung medical center, Seoul, Republic of Korea
  More Information

Responsible Party: Green Cross Corporation Identifier: NCT01645189     History of Changes
Other Study ID Numbers: GC1111C 
Study First Received: July 16, 2012
Last Updated: July 7, 2014
Health Authority: Korea: Food and Drug Administration

Keywords provided by Green Cross Corporation:
Hunter syndrome

Additional relevant MeSH terms:
Mucopolysaccharidosis II
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Intellectual Disability
Lysosomal Storage Diseases
Mental Retardation, X-Linked
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations processed this record on May 24, 2016