Hunter Syndrome
Conditions
Keywords
Hunter syndrome, Mucopolysaccharidosis II, Hunterase
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
Interventions
None listed
Sponsors
Green Cross Corporation
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse event and adverse drug reaction. | Once a week up to 5years |
Secondary
| Measure | Time frame |
|---|---|
| Change of vital sign, physical and clinical examination, anti-idurasulfase-beta antibody status. | Base line and every three months up to 5years.(exception: clinical examination-> baseline and every six months up to 5years) |
Other
| Measure | Time frame |
|---|---|
| Percent change of urine GAG. | Baseline and every three months up to 5years. |
| Percent change of 6 minute walking test. | Baseline and every 6 months up to 5years. |
Countries
South Korea
Contacts
Primary ContactDong-Kyu Jin
Outcome results
None listed