Vyndaqel Capsules Special Investigation -Investigation on Long-term Use-
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 500
- 主要终点
- Percentage of Participants With ADRs in This Study
研究概览
简要总结
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received this drug under actual use conditions after its marketing.
详细描述
Both prospective and retrospective is acceptable All the patients whom an investigator prescribes Vyndaqel should be registered
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The subjects of this surveillance are all patients who received Vyndaqel.
排除标准
- •Patients not receive Vyndaqel.
研究组 & 干预措施
Vyndaqel
干预措施: Vyndaqel (Drug)
结局指标
主要结局
Percentage of Participants With ADRs in This Study
时间窗: 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)
An adverse drug reaction (ADR) was any untoward medical occurrence attributed to Vyndaqel capsules 20mg in a participant who received Vyndaqel capsules 20mg. A serious adverse drug reaction (SADR) was ADR resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect. Relatedness to Vyndaqel capsules 20mg was assessed by the physician.
次要结局
- Change From Baseline in the NIS-LL Score(Week 78, Week 156)
- Change From Baseline in the TQOL Score of the Norfolk QOL-DN(Week 78, Week 156)
- Change From Baseline in mBMI(Week 26, Week 52, Week 78, Week 104, Week 130, Week 156)
- Time Course of Ambulatory Status(Week 156)
- Change From Baseline in the Total Neurological Assessment Score(Week 52, Week 104, Week 156)
- Assessment of Survival Time for Death From Any Cause(3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.))
- Assessment of Survival Time for Death Associated With ATTR-PN(3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.))