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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Hemophilia

Intervention / Treatment
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* Other: Qualitative interview


Participation Criteria
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Eligibility Criteria
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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a member of their family People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family People with haemophilia A or B who are definitely not interested in or unaware of gene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.

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 100 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Early dose finding cohort<br>People with Haemophilia who took part in the early dose finding studies | Other: Qualitative interview<br>* Qualitative semi structured interview<br>|
| Subsequent studies group cohort<br>People with haemophilia who took part in subsequent gene therapy studies | Other: Qualitative interview<br>* Qualitative semi structured interview<br>|
| Withdrawn/ineligible Cohort<br>People with haemophilia who were withdrawn or proved ineligible | Other: Qualitative interview<br>* Qualitative semi structured interview<br>|
| Not interested cohort<br>People with haemophilia who are definitely not interested in gene therapy | Other: Qualitative interview<br>* Qualitative semi structured interview<br>|
| Not offered cohort<br>People with haemophilia who are interested in gene therapy but have not been the opportunity to take part | Other: Qualitative interview<br>* Qualitative semi structured interview<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| patient expectations | To explore the expectations that patients, and families in the UK have of gene therapy and its position in contemporary and future haemophilia management | 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 | 

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Haemophilia, Gene Therapy, Quality of Life