Clinical trial NCT04723680: An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
Official title: An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia
Keywords: Haemophilia, Gene Therapy, Quality of Life
Lead sponsor: Haemnet
Sponsor class: OTHER
Collaborators: name: UniQure Biopharma B.V.
First posted: 2021-01-26
Enrollment: 69
Sex: MALE
Minimum age (years): 16
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: People with haemophilia and a member of their family

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.

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* People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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

Primary outcomes:
measure: patient expectations
timeFrame: 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 outcomes:
measure: Lived experience
timeFrame: 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
measure: Impact of gene therapy
timeFrame: 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
measure: Impact of ineligibility
timeFrame: 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
measure: Impact of withdrawal
timeFrame: 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
measure: Why not interested
timeFrame: 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
Other outcomes:
Locations:
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust
state: Oxfordshire
Study officials:
affiliation: Researcher
name: Simon P Fletcher
role: PRINCIPAL_INVESTIGATOR
References: