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

Hemophilia Clinical Trial

— Exigency  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04723680
• Other study ID #: v3Nov2020
• Status: Completed
• Start date: August 13, 2020
• Est. completion date: February 28, 2023

Study information

• Verified date: February 2023
• Source: Haemnet
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 69
• Est. completion date: February 28, 2023
• Est. primary completion date: February 28, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 16 Years to 100 Years

Eligibility

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• People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A

Intervention

Other:
• Qualitative interview
Qualitative semi structured interview

Locations

Country	Name	City	State
United Kingdom	Oxford University Hospitals NHS Foundation Trust	Oxford	Oxfordshire

Sponsors (2)

Lead Sponsor	Collaborator
Haemnet	UniQure Biopharma B.V.

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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