NCT07344376已完成3 期
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) in Patients With Hunter Syndrome
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Laboratory tests (CBC, Chemistry, Urinanalysis)
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subject diagnosed with Hunter syndrome (MPS II) who completed activities at the EOS visit (Visit 54) in the GC1111_P3 study
- •(Subject who did not participate in the GC1111_P3 study) Subject diagnosed with Hunter syndrome (MPS II) who received Hunterase for more than 6 months
- •(Subjects who did not participate in the GC1111_P3 study) Males aged ≥ 5
- •Informed consent form voluntarily signed by the subject or by a legally acceptable representative
- •Subject who agrees to use contraception
排除标准
- •Subject with hypersensitivity to any of the ingredients of the investigational product
- •Subject impossible to perform follow-up observation of the safety
- •Subject who received the treatment with another investigational product within 14 days prior to the start of study drug
- •Subject who plans to be treated with another investigational product during the study period
- •Subject who has history of tracheostomy, bone marrow transplant, or cord blood transplant
- •Any other inappropriate conditions for study participation at the investigator's discretion
研究组 & 干预措施
Hunterase
Other
Single arm
干预措施: Hunterase (Drug)
结局指标
主要结局
Laboratory tests (CBC, Chemistry, Urinanalysis)
时间窗: Every visit until 12months
Abnormality of Laboratory tests results
Physical examination
时间窗: Every visit until 12months
Abnormality of physical examination
Adverse Event
时间窗: Every visit until 12months
occurence, grade
Electrocardiography
时间窗: Every 6 months until 12months
Abnormality of ECG results
次要结局
- 6-minute walk test(Every 4months until 12months)
- Urine GAG (Heparan sulfate, Dermatan sulfate)(Every visit until 12months)
研究者
研究点 (1)
Loading locations...
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