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Safety and Efficacy of Hunterase (GC1111)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01645189
Recruitment Status : Completed
First Posted : July 20, 2012
Last Update Posted : July 8, 2014
Information provided by (Responsible Party):
Green Cross Corporation

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.

Condition or disease Intervention/treatment Phase
Hunter Syndrome Biological: Hunterase Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : July 2012
Actual Primary Completion Date : September 2013
Actual Study Completion Date : September 2013

Arm Intervention/treatment
Experimental: Test drug
Biological: Hunterase
once weekly, 0.5mg/kg IV infusion

Primary Outcome Measures :
  1. Incidence of adverse events [ Time Frame: One year ]

Secondary Outcome Measures :
  1. change of anti-idursulfase-beta antibody status [ Time Frame: baseline and one year ]
  2. Percent Change of Urine GAG [ Time Frame: baseline to 53 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes 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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01645189

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

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Responsible Party: Green Cross Corporation Identifier: NCT01645189     History of Changes
Other Study ID Numbers: GC1111C
First Posted: July 20, 2012    Key Record Dates
Last Update Posted: July 8, 2014
Last Verified: July 2014
Keywords provided by Green Cross Corporation:
Hunter syndrome
Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases