Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 5
- 试验地点
- 6
- 主要终点
- Change From Baseline in Weight Over One Year Study Period
研究概览
简要总结
The purpose of this non-interventional, observational study which is conducted in Mexico is to evaluate the safety profile of elaprase (idulsurfase) in participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants of both genders
- •Participants to whom idursulfase (Elaprase) has been prescribed
- •Participants with Hunter syndrome
- •Participants accepting to participate
- •Participants signing the informed consent and privacy notice for handling personal information in: the program Contigo Shire (training, diagnosis, family trees) and the intensive pharmacovigilance study for Elaprase. If the participant is unable to provide legal consent (example: under legal age), the participant's legal guardian must provide consent
排除标准
- •Participants withdrawing their consent to continue participating in the non-intervention intensive pharmacovigilance study for Elaprase
- •Known hypersensitivity or presence of any contraindication to Elaprase that cannot be controlled by pre-medication
- •A physical condition that would not permit intravenous administration of enzyme replacement therapy (ERT)
- •Participants enrolled in Shire sponsored Elaprase Registry or any other Shire sponsored Elaprase study
结局指标
主要结局
Change From Baseline in Weight Over One Year Study Period
时间窗: Baseline, 12 months
Change in weight over one year study period will be assessed.
Incidence of Adverse Events According to the System Organ Class and MedDRA Preferred Term
时间窗: During 12 months of study participation
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that do not necessarily have a causal relationship with this treatment. Incidence of adverse events according to the system organ class and MedDRA preferred term will be reported.
Incidence of Adverse Events During Administration of Elaprase
时间窗: During 12 months of study participation
An adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that do not necessarily have a causal relationship with this treatment. Incidence of adverse events during administration of elaprase will be reported.
Change From Baseline in Height Over One Year Study Period
时间窗: Baseline, 12 months
Change in height over one year study period will be assessed.
Incidence of Adverse Events 2 months After Early Termination of Elaprase Treatment.
时间窗: 2 months after early termination of Elaprase treatment
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that do not necessarily have a causal relationship with this treatment. Incidence of adverse events 2 months after early termination of Elaprase treatment will be reported.
次要结局
未报告次要终点