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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Test drug | Experimental | Idursulfase-beta |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hunterase | Biological | once weekly, 0.5mg/kg IV infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events | One year |
| Measure | Description | Time Frame |
|---|---|---|
| change of anti-idursulfase-beta antibody status | baseline and one year | |
| Percent Change of Urine GAG | baseline to 53 weeks |
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Inclusion Criteria:
The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:
as measured in plasma, leukocytes, or fibroblasts,
That corresponds to one or more of the following:
< 6 years old and male
Patients who are able to comply with the study requirements
The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Dong-Kyu Jin | Samsung medical center, Seoul, Republic of Korea | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Samsug Medical Center | Seoul | South Korea |
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| ID | Term |
|---|---|
| D016532 | Mucopolysaccharidosis II |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |