An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 69
- 试验地点
- 1
- 主要终点
- patient expectations
研究概览
简要总结
This study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people and families affected by haemophilia who have been or will be exposed to gene therapy.
详细描述
This is a prospective observational multiple cohort qualitative research study to be conducted among diverse groups within the haemophilia community whose lives may have been impacted by gene therapy.
The study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and offer insight into how these patients and families cope with haemophilia.
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
- 16 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •People with haemophilia A or B who consented to and have undergone gene therapy in the early dose-finding studies and a member of their family
- •People with haemophilia A or B who consented to a gene therapy trial following the results of the early studies and a member of their family
- •People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family
- •People with haemophilia A or B who are definitely not interested in or unaware of gene therapy and a member of their family
- •People with haemophilia A or B who are interested in but have not been offered gene therapy
- •Those who have given written consent to be in the study
- •All participants will be ≥16 years.
排除标准
- •Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.
结局指标
主要结局
patient expectations
时间窗: Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach
To explore the expectations that patients, and families in the UK have of gene therapy and its position in contemporary and future haemophilia management
次要结局
- Lived experience(Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach)
- Impact of ineligibility(Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach)
- Impact of withdrawal(Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach)
- Why not interested(Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach)
- Impact of gene therapy(Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach)