This study aimed to examine the real-world experience and impact of gene therapy on people and families affected by haemophilia. No treatment is given; information is gathered through qualitative interviews. Participants included people with haemophilia A or B who had undergone gene therapy in early dose-finding studies, or who consented to a gene therapy trial following those results, along with a member of their family.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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a member of their family * People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family * People with haemophilia A or B who are definitely not interested in or unaware of gene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.