HIT study (HIT study)

Not yet recruiting

• Conditions: Mucopolysaccharidosis Type II

• Registration Number: jRCT1030250124
• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-05-23
• Last Update Posted: 2025-05-26

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 30

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

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)

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Anthropometric measurements	from the start of observation to 104 weeks	Height/weight/head circumference
Visual evaluation	from the start of observation to 104 weeks	Electroretinography (ERG)/intraocular pressure testing/funduscopy/optical coherence tomography (OCT) testing/simple vision testing
Auditory evaluation	from the start of observation to 104 weeks	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).
Evaluation of respiratory function	from the start of observation to 104 weeks	Fiberpharyngeal examination/chest computed tomography (CT)/respiratory infection symptoms
Evaluation of cardiovascular function	from the start of observation to 104 weeks	Echocardiographic /BNP precursor N-terminal fragment (NT-proBNP) (brain natriuretic peptide (BNP) is also acceptable if difficult to measure NT-proBNP)