An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families

Completed

• Conditions: Hemophilia
• Interventions: Other: Qualitative interview

• Registration Number: NCT04723680
• Lead Sponsor: Haemnet

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2020-08-13
• Study First Submitted: 2021-01-15
• Study First Submitted QC: 2021-01-20
• Study First Posted: 2021-01-26
• Status Verified: 2023-02
• Primary Completion: 2023-02-28
• Study Completion: 2023-02-28
• Last Update Submitted: 2023-02-28
• Last Update Posted: 2023-03-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 69

Inclusion Criteria

• 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• People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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.

Exclusion Criteria

• Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Not interested cohort	Qualitative interview	People with haemophilia who are definitely not interested in gene therapy
Not offered cohort	Qualitative interview	People with haemophilia who are interested in gene therapy but have not been the opportunity to take part
Subsequent studies group cohort	Qualitative interview	People with haemophilia who took part in subsequent gene therapy studies
Withdrawn/ineligible Cohort	Qualitative interview	People with haemophilia who were withdrawn or proved ineligible
Early dose finding cohort	Qualitative interview	People with Haemophilia who took part in the early dose finding studies

Primary Outcome Measures

Name	Time	Method
patient expectations	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

Secondary Outcome Measures

Name	Time	Method
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	To understand the lived experience of people who have undergone gene therapy
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	To understand the impact of ineligibility for gene therapy trials
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	To understand the impact of withdrawal from gene therapy on individuals and their ongoing attitude to their haemophilia care
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	To understand why some patients and families opt not to participate in gene therapy trials as a treatment option
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	To understand the as yet "unseen" impact of gene therapy on the extended family

Trial Locations

Locations (1)

Oxford University Hospitals NHS Foundation Trust; 🇬🇧 Oxford, Oxfordshire, United Kingdom