In-trial Qualitative Interviews to Explore the Impact of Gene Therapy in Hemophilia A & B
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- Pfizer
- 入组人数
- 40
- 主要终点
- Frequency of self-reported symptoms pre and post hemophilia gene therapy
研究概览
简要总结
The purpose of this study is to learn about experiences of patients with hemophilia A and B after taking gene therapy. The experiences of patients will be studied through online interviews.
This study is seeking participants who are:
- part of the Pfizer's gene therapy clinical studies or
- in the long-term follow up for these clinical programs. Participants will have one study visit at the clinic and one online interview. The planned duration for each participant will be 1 to 2 months. This covers the time from entering the study to end of the online interview.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participation in the Pfizer's gene therapy clinical trials for Hemophilia A or B .
- •Participants will be one year or more post-gene therapy
- •Participants will be willing to provide written consent and will be willing to comply with the study requirements
排除标准
- •No exclusion criteria
结局指标
主要结局
Frequency of self-reported symptoms pre and post hemophilia gene therapy
时间窗: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Frequency of self-reported severity pre and post hemophilia gene therapy
时间窗: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Frequency of self-reported impacts pre and post hemophilia gene therapy
时间窗: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
Change in the proportion of patients answering 5-point Likert scale questions about the burden of hemophilia on the participant's life pre-post gene therapy
时间窗: From date of dosing in the gene therapy clinical trial until the interview date, no less than 52 weeks.
次要结局
未报告次要终点