Status:
COMPLETED
An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
Lead Sponsor:
Haemnet
Collaborating Sponsors:
UniQure Biopharma B.V.
Conditions:
Hemophilia
Eligibility:
MALE
16-100 years
Brief 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...
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....
Eligibility Criteria
Inclusion
- 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- People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family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
- Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.
Key Trial Info
Start Date :
August 13 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 28 2023
Estimated Enrollment :
69 Patients enrolled
Trial Details
Trial ID
NCT04723680
Start Date
August 13 2020
End Date
February 28 2023
Last Update
March 1 2023
Active Locations (1)
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1
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, United Kingdom, OX3 7LE