Post Trial Access for Study SHP677-304: Recombinant Von Willebrand Factor (rVWF) for Adult and Pediatric Subjects With Severe Von Willebrand Disease (VWD)
试验速览
- 阶段
- 不适用
- 状态
- Available
- 发起方
研究概览
简要总结
The post-trial access program allows eligible participants to gain access to unlicensed treatment on compassionate grounds. Recombinant von Willebrand factor (rVWF) also known as TAK-577, is a medicine to help treat Von Willebrand Disease (VWD). This post-trial access program enables continued access to children and adults who are benefitting from treatment on study SHP677-304 (NCT03879135) study.
详细描述
This is a post-trial access program in which the drug being given is called TAK-577. This study will provide access to TAK-577 before marketing authorization for eligible participants with severe VWD who are benefitting from treatment on study SHP677-304 and cannot adequately be treated via the current standard of care and cannot enter a clinical trial.
All participants will receive TAK-577 as a sequential intravenous infusion based on their weight.
This is a multi-center, international program. Participants will continue treatment until a benefit is no longer derived from the treatment (or treatment is no longer tolerable), the sponsor decision to end the program, the participant chooses to discontinue the treatment, or TAK-577 becomes commercially available for children.
研究设计
- 研究类型
- Expanded Access
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant has completed the treatment period of the SHP677-304 study (at least 12 months on study treatment).
- •Participant had good clinical response to rVWF treatment.
- •Participant does not have access to any comparable or satisfactory alternative replacement therapy available at country level.
- •Participant will be/has been negatively impacted by discontinuation of rVWF.
- •Participant and/or a parent(s)/legal guardian is informed of the nature of the post-trial access program and can provide written informed consent for themselves or the child to participate (with assent from a child when appropriate) before treatment).
排除标准
- •1. Participants with known hypersensitivity/intolerance to the study drug will not be eligible for this study