Brief Title
An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
Official Title
An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
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 therapy.
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
Primary Outcome
patient expectations
Secondary Outcome
Lived experience
Condition
Hemophilia
Intervention
Qualitative interview
Study Arms / Comparison Groups
Early dose finding cohort
Description: People with Haemophilia who took part in the early dose finding studies
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
69
Start Date
August 13, 2020
Completion Date
February 28, 2023
Primary Completion Date
February 28, 2023
Eligibility Criteria
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a member of their family - People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family - People with haemophilia A or B who are definitely not interested in or unaware of gene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.
Gender
Male
Ages
16 Years - 100 Years
Accepts Healthy Volunteers
No
Contacts
Simon P Fletcher, ,
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT04723680
Organization ID
v3Nov2020
Responsible Party
Sponsor
Study Sponsor
Haemnet
Collaborators
UniQure Biopharma B.V.
Study Sponsor
Simon P Fletcher, Principal Investigator, Researcher
Verification Date
February 2023