Hunter syndrome IZCARGO Treatment study ( HIT study )
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 30
- 主要终点
- Visual evaluation
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- No limit 至 No limit（—）
入选标准
- •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)
- •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
- •Patients undergoing or scheduled to undergo ERT with pabinafusp alfa (genetic recombination) or idursulfase (genetic recombination)
排除标准
- •Patients receiving enzyme preparations despite a history of anaphylactic shock who are receiving the enzyme preparations.
- •Patients who have received investigational products other than enzyme perparations for currently marketed for MPS II
- •Patients receiving idulfase beta (genetic recombination)
- •Patients who have undergone hematopoietic stem cell transplantation in the past (however, patients with engraftment failure are eligible for registration)
结局指标
主要结局
Visual evaluation
时间窗: from the start of observation to 104 weeks
Electroretinography (ERG)/intraocular pressure testing/funduscopy/optical coherence tomography (OCT) testing/simple vision testing
Auditory evaluation
时间窗: 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
时间窗: from the start of observation to 104 weeks
Fiberpharyngeal examination/chest computed tomography (CT)/respiratory infection symptoms
Evaluation of cardiovascular function
时间窗: 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)
Anthropometric measurements
时间窗: from the start of observation to 104 weeks
Height/weight/head circumference
次要结局
未报告次要终点