臨床研究等提出・公開システム

Hunter syndrome IZCARGO Treatment study ( HIT study )

HIT study (HIT study)

Yoshida Pascal

JCR Pharmaceuticals Co., Ltd.

3-19 Kasuga-cho, Ashiya, Hyogo, Japan

hit_study@jp.jcrpharm.com

Noguchi Atsushi

JCR Pharmaceuticals Co., Ltd.

3-19 Kasuga-cho, Ashiya, Hyogo, Japan

+81-797-32-8582

hit_study@jp.jcrpharm.com

Pending

June. 09, 2025

Observational

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 undergoing or scheduled to undergo ERT with pabinafusp alfa (genetic recombination) or idursulfase (genetic recombination)

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)

Mucopolysaccharidosis Type II

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

+81-3-6416-1868

Mar. 06, 2025

No

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