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

Haemnet

Status: Completed

Conditions: Hemophilia

Treatments: Other: Qualitative interview

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT04723680

v3Nov2020

Details and patient eligibility

About

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.

Full 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.

Enrollment: 69 patients

Sex: Male

Ages: 16 to 100 years old

Volunteers: No Healthy Volunteers

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• People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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

Trial design

69 participants in 5 patient groups

Early dose finding cohort

Description:

People with Haemophilia who took part in the early dose finding studies

Treatment:

Other: Qualitative interview

Subsequent studies group cohort

Description:

People with haemophilia who took part in subsequent gene therapy studies

Treatment:

Other: Qualitative interview

Withdrawn/ineligible Cohort

Description:

People with haemophilia who were withdrawn or proved ineligible

Treatment:

Other: Qualitative interview

Not interested cohort

Description:

People with haemophilia who are definitely not interested in gene therapy

Treatment:

Other: Qualitative interview

Not offered cohort

Description:

People with haemophilia who are interested in gene therapy but have not been the opportunity to take part

Treatment:

Other: Qualitative interview