The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Green Cross Corporation
Information provided by (Responsible Party):
Green Cross Corporation Identifier:
First received: January 19, 2014
Last updated: June 19, 2014
Last verified: June 2014
The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Hunter Syndrome

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Long-term Safety of Hunterase (Idursulfase-beta) in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Resource links provided by NLM:

Further study details as provided by Green Cross Corporation:

Primary Outcome Measures:
  • Incidence of adverse event and adverse drug reaction. [ Time Frame: Once a week up to 5years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change of vital sign, physical and clinical examination, anti-idurasulfase-beta antibody status. [ Time Frame: Base line and every three months up to 5years.(exception: clinical examination-> baseline and every six months up to 5years) ] [ Designated as safety issue: Yes ]

Other Outcome Measures:
  • Percent change of urine GAG. [ Time Frame: Baseline and every three months up to 5years. ] [ Designated as safety issue: No ]
  • Percent change of 6 minute walking test. [ Time Frame: Baseline and every 6 months up to 5years. ] [ Designated as safety issue: No ]

Estimated Enrollment: 34
Study Start Date: January 2014
Estimated Study Completion Date: January 2020
Estimated Primary Completion Date: January 2020 (Final data collection date for primary outcome measure)

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Hunter Syndrome(Mucopolysaccharidosis II) Patients

Inclusion Criteria:

  • Patients who have a diagnosis of Hunter syndrome(Mucopolysaccharidosis II).
  • Patients who are administered idurasulfase-beta or willing to be administered idurasulfase-beta.
  • Patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study.

Exclusion Criteria:

  • Patients who have participated in any other blind clinical trials.
  • Patient who cannot be tracked about safety.
  • Patients who are judged disqualified to participate clinical trials by investigator for other causes.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02044692

Contact: Dong-Kyu Jin

Korea, Republic of
Samsung Medical Center Recruiting
Seoul, Korea, Republic of
Contact: Dongkyu Jin         
Principal Investigator: Dong Kyu Jin         
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: NCT02044692     History of Changes
Other Study ID Numbers: GC1111_OS 
Study First Received: January 19, 2014
Last Updated: June 19, 2014
Health Authority: Korea: Ministry of Food and Drug Safety

Keywords provided by Green Cross Corporation:
Hunter syndrome
Mucopolysaccharidosis II

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
Pathologic Processes processed this record on May 26, 2016