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Completed

NCT Number: NCT04723680

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

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.

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Key information

Conditions

Hemophilia Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Age range

16 year–100 year

Sex eligibility

Male

Study type

Observational

Primary location

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OX3 7LE, United Kingdom

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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  • People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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

Treatment and study plan

Qualitative interview

Other

Qualitative semi structured interview

Primary outcomes

  1. patient expectations

    Time frame: 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 outcomes

  1. Lived experience

    Time frame: 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

  2. Impact of gene therapy

    Time frame: 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

  3. Impact of ineligibility

    Time frame: 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

  4. Impact of withdrawal

    Time frame: 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

  5. Why not interested

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Haemnet

Other

Collaborators

  • UniQure Biopharma B.V.

Registry information

Acronym: Exigency

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Jan 26, 2021
Registry last updated
Mar 1, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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