An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
NCT ID: NCT04723680
Last Updated: 2023-03-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
69 participants
OBSERVATIONAL
2020-08-13
2023-02-28
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Early dose finding cohort
People with Haemophilia who took part in the early dose finding studies
Qualitative interview
Qualitative semi structured interview
Subsequent studies group cohort
People with haemophilia who took part in subsequent gene therapy studies
Qualitative interview
Qualitative semi structured interview
Withdrawn/ineligible Cohort
People with haemophilia who were withdrawn or proved ineligible
Qualitative interview
Qualitative semi structured interview
Not interested cohort
People with haemophilia who are definitely not interested in gene therapy
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
Qualitative interview
Qualitative semi structured interview
Interventions
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Qualitative interview
Qualitative semi structured interview
Eligibility Criteria
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Inclusion Criteria
* 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* People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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
16 Years
100 Years
MALE
No
Sponsors
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UniQure Biopharma B.V.
INDUSTRY
Haemnet
OTHER
Responsible Party
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Principal Investigators
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Simon P Fletcher
Role: PRINCIPAL_INVESTIGATOR
Researcher
Locations
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Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Other Identifiers
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v3Nov2020
Identifier Type: -
Identifier Source: org_study_id
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