Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 316
- 试验地点
- 71
- 主要终点
- Percentage of Participants With Adverse Events
研究概览
简要总结
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety, effectiveness, and prescription patterns in routine clinical practice.
详细描述
This is a multicenter, observational study of participants receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 360 participants across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include participants who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) and who provide consent. Participants will be followed for at least 5 years from their date of enrollment. Safety and efficacy data will be collected in conjunction with routine visits conducted as per usual care. Although there are no protocol-mandated procedures, it is expected that physicians and other caregivers will follow published treatment guidelines and standards of care.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program)
- •Willing to provide written informed consent to allow the study data collection procedures (either by the participant or through authorisation by a legal guardian)
排除标准
- •Participants who are receiving ataluren or placebo in a blinded, randomized clinical trial, or ataluren in any other ataluren clinical trial or cohort early access program that prevents participation in this study
研究组 & 干预措施
Translarna
Participants with nmDMD receiving Translarna will be followed for at least 5 years from their date of enrollment, or until participant withdrawal of consent or death, whichever occurs first.
结局指标
主要结局
Percentage of Participants With Adverse Events
时间窗: 5 years
次要结局
- Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling(5 years)