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

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ClinicalTrials.gov Identifier: NCT02044692
Recruitment Status : Recruiting
First Posted : January 24, 2014
Last Update Posted : June 20, 2014
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation

Brief Summary:
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

Condition or disease
Hunter Syndrome

Study Type : Observational
Estimated Enrollment : 34 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Long-term Safety of Hunterase (Idursulfase-beta) in Hunter Syndrome(Mucopolysaccharidosis II) Patients
Study Start Date : January 2014
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2020





Primary Outcome Measures :
  1. Incidence of adverse event and adverse drug reaction. [ Time Frame: Once a week up to 5years ]

Secondary Outcome Measures :
  1. 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) ]

Other Outcome Measures:
  1. Percent change of urine GAG. [ Time Frame: Baseline and every three months up to 5years. ]
  2. Percent change of 6 minute walking test. [ Time Frame: Baseline and every 6 months up to 5years. ]


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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Hunter Syndrome(Mucopolysaccharidosis II) Patients
Criteria

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.

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 ClinicalTrials.gov identifier (NCT number): NCT02044692


Contacts
Contact: Dong-Kyu Jin jindk.jin@samsung.com

Locations
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
Investigators
Principal Investigator: Dong-Kyu Jin Samsung medical center, Seoul, Republic of Korea

Responsible Party: Green Cross Corporation
ClinicalTrials.gov Identifier: NCT02044692     History of Changes
Other Study ID Numbers: GC1111_OS
First Posted: January 24, 2014    Key Record Dates
Last Update Posted: June 20, 2014
Last Verified: June 2014

Keywords provided by Green Cross Corporation:
Hunter syndrome
Mucopolysaccharidosis II
Hunterase

Additional relevant MeSH terms:
Syndrome
Mucopolysaccharidoses
Mucopolysaccharidosis II
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
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