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

February 28, 2023 updated by: Haemnet
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

69

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United Kingdom
    • Oxfordshire
      • Oxford, Oxfordshire, United Kingdom, OX3 7LE
        • Oxford University Hospitals NHS Foundation Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 100 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

People with haemophilia and a member of their family

Description

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  • People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Early dose finding cohort
People with Haemophilia who took part in the early dose finding studies
Other: Qualitative interview
Qualitative semi structured interview
Subsequent studies group cohort
People with haemophilia who took part in subsequent gene therapy studies
Other: Qualitative interview
Qualitative semi structured interview
Withdrawn/ineligible Cohort
People with haemophilia who were withdrawn or proved ineligible
Other: Qualitative interview
Qualitative semi structured interview
Not interested cohort
People with haemophilia who are definitely not interested in gene therapy
Other: Qualitative interview
Qualitative semi structured interview
Not offered cohort
People with haemophilia who are interested in gene therapy but have not been the opportunity to take part
Other: Qualitative interview
Qualitative semi structured interview

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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
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
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
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
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
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
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
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
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Haemnet

Collaborators

UniQure Biopharma B.V.

Investigators

  • Principal Investigator: Simon P Fletcher, Researcher

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 13, 2020

Primary Completion (Actual)

February 28, 2023

Study Completion (Actual)

February 28, 2023

Study Registration Dates

First Submitted

January 15, 2021

First Submitted That Met QC Criteria

January 20, 2021

First Posted (Actual)

January 26, 2021

Study Record Updates

Last Update Posted (Actual)

March 1, 2023

Last Update Submitted That Met QC Criteria

February 28, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • v3Nov2020

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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