Working... Menu

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

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: NCT01301898
Recruitment Status : Completed
First Posted : February 23, 2011
Last Update Posted : February 14, 2012
Information provided by (Responsible Party):
Green Cross Corporation

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

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis II Drug: GC1111_0.5mg/kg Drug: GC1111_1.0mg/kg Drug: Elaprase_0.5mg/kg Phase 1 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
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 Start Date : May 2010
Actual Primary Completion Date : March 2011
Actual Study Completion Date : March 2011

Arm Intervention/treatment
Experimental: GC1111_0.5mg/kg Drug: GC1111_0.5mg/kg
Experimental: GC1111_1.0mg/kg Drug: GC1111_1.0mg/kg
Active Comparator: Elaprase_0.5mg/kg Drug: Elaprase_0.5mg/kg

Primary Outcome Measures :
  1. Primary Outcome [ Time Frame: baseline, every 4 weeks ]
    measurement of % change of Urine GAG

Secondary Outcome Measures :
  1. Secondary Outcome [ Time Frame: baseline, every 12 weeks ]
    • 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   6 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

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): NCT01301898

Layout table for location information
Korea, Republic of
Samsung Medical Center
Seoul, Irwon-dong, Gangnam-Gu, Korea, Republic of, 135-710
Sponsors and Collaborators
Green Cross Corporation

Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Green Cross Corporation Identifier: NCT01301898     History of Changes
Other Study ID Numbers: GC1111_P1/2
First Posted: February 23, 2011    Key Record Dates
Last Update Posted: February 14, 2012
Last Verified: February 2012

Keywords provided by Green Cross Corporation:
Hunter syndrome

Additional relevant MeSH terms:
Layout table for MeSH terms
Mucopolysaccharidosis II
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System