Mucopolysaccharidosis Type II
Conditions
Interventions
None listed
Sponsors
Yamamoto Tatsuyoshi
Eligibility
Inclusion criteria
Inclusion criteria: 1)Patients with whom written consent can be obtained patients younger than 18 years of age at the time of obtaining informed consent or patients whose intention cannot be confirmed due to symptoms associated with MPS II may obtain written consent from the legally acceptable representative (provided, however, that written consent shall be obtained from the assignee whenever feasible) 2)Patients who have been comprehensively diagnosed with MPS II based on decreased iduronate-2-sulfatase activity or genetic analysis of leukocytes, plasma, or cultured skin fibroblasts 3)Patients participating in post marketing studies or use results survey of pabinafusp alfa (genetical recombination), or those undergoing ERT with idursulfase (genetical recombination)
Exclusion criteria
Exclusion criteria: 1)Patients receiving enzyme preparations despite a history of anaphylactic shock who are receiving the enzyme preparations. 2)Patients who have received investigational products other than enzyme perparations for currently marketed for MPS II 3)Patients receiving idulfase beta (genetic recombination) 4)Patients who have undergone hematopoietic stem cell transplantation in the past (however, patients with engraftment failure are eligible for registration)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Changes in the following items from the start of observation to 104 weeks (1) Visual evaluation Electroretinography (ERG)/intraocular pressure testing/funduscopy/optical coherence tomography (OCT) testing/simple vision testing (2) Auditory evaluation Auditory steady response test (ASSR) (auditory brainstem response audiometry (ABR) can also be performed if it is difficult to perform ASSR)/otitis media symptoms/tympanostomy tube use/hearing aid use/standard pure tone audiometry (PTA) or conditioned orienting response audiometry (COR). (3) Evaluation of respiratory function Fiberpharyngeal examination/chest computed tomography (CT)/respiratory infection symptoms (4) Evaluation of cardiovascular function Echocardiographic /BNP precursor N-terminal fragment (NT-proBNP) (brain natriuretic peptide (BNP) is also acceptable if difficult to measure NT-proBNP) (5) Anthropometric measurements Height/weight/head circumference | — |
Contacts
Public ContactAtsushi Noguchi
JCR Pharmaceuticals Co., Ltd.
Outcome results
None listed