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| Name | Class |
|---|---|
| Samsung Medical Center | OTHER |
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The objective of this study is to assess the long-term safety and efficacy of Hunterase for approximately 1 year in subjects who completed the end of study (EOS) visit (Visit 54) tests in the Phase 3 Hunterase study and patients who received Hunterase for more than 6 months
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hunterase | Other | Single arm |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hunterase | Drug | 0.5mg/kg weekly IV administration |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Laboratory tests (CBC, Chemistry, Urinanalysis) | Abnormality of Laboratory tests results | Every visit until 12months |
| Physical examination | Abnormality of physical examination | Every visit until 12months |
| Adverse Event | occurence, grade | Every visit until 12months |
| Electrocardiography | Abnormality of ECG results | Every 6 months until 12months |
| Measure | Description | Time Frame |
|---|---|---|
| 6-minute walk test | Mean, Change and percent change of 6-minute walk test | Every 4months until 12months |
| Urine GAG (Heparan sulfate, Dermatan sulfate) | Mean, Change and percent change of Urine HS/DS |
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Inclusion Criteria:
Exclusion Criteria:
Male
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Samsung Medical Center | Seoul | South Korea |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42421116 | Derived | Im M, Song A, Park H, Kim J, Kim C, Noh ES, Bae GY, Sung J, Hwang J, Ahn YJ, Kim H, Oh Y, Kim B, Cho SY. Sustained clinical benefit of idursulfase beta in mucopolysaccharidosis II: two-year experience from a phase 3 extension study including patients switched from idursulfase. Orphanet J Rare Dis. 2026 Jul 9. doi: 10.1186/s13023-026-04492-2. Online ahead of print. |
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| ID | Term |
|---|---|
| D013398 | Sudden Infant Death |
| D016532 | Mucopolysaccharidosis II |
| ID | Term |
|---|---|
| D003645 | Death, Sudden |
| D003643 | Death |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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Phase 3 Extension study
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| Every visit until 12months |
| D066088 |
| Infant Death |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| 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 |