Hemophilia
Conditions
Keywords
Haemophilia, Gene Therapy, Quality of Life
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.
Interventions
OTHERQualitative interview
Qualitative semi structured interview
Sponsors
UniQure Biopharma B.V.
Haemnet
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
16 Years to 100 Years
Healthy volunteers
No
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a member of their family * People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family * People with haemophilia A or B who are definitely not interested in or unaware of gene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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 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 |
| 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 |
Countries
United Kingdom
Outcome results
None listed