Survey of quality of life in adults with haemophilia A
Research type
Research Study
Full title
Quality of life in adults with haemophilia A: a UK centre survey
IRAS ID
252219
Contact name
Simon Fletcher
Contact email
Sponsor organisation
BioMarin UK, Limited
Duration of Study in the UK
0 years, 4 months, 31 days
Research summary
This is a questionnaire-based study of health-related quality of life (HRQOL) in adult males (18+ years) with haemophilia A, conducted in haemophilia treatment centres and comprehensive care centres in the UK. Participants will complete a paper-based questionnaire booklet consisting of six patient-reported outcome (PRO) measures, supplemented with a small number of additional targeted questions.
The primary aim of this study is to generate contemporary (HRQOL) data among a UK cohort of patients with haemophilia through use of generic and disease-specific PRO measures. This information will allow for the future objectives of: assessing the level of conceptual overlap between disease-specific and generic measures of HRQOL; and assessing the feasibility of mapping between PRO measures for use in future evaluations of HRQOL.
A total of n=200 participants (no active inhibitors) are sought; an additional sample of n=20 patients with an active inhibitor will also be sought. Participant recruitment is expected to be open for up to 20 weeks. Participants will be provided with an information leaflet and informed consent form (ICF) in order to guide their decision to participate in the study. Participants will be reimbursed with a gift voucher for their time. It is intended that this questionnaire will be completed prior to or following a routine consultation, with an estimated completion time of 40 minutes.
The study is sponsored and funded by BioMarin (U.K.) Ltd. Data entry (pseudonymised) and analysis will be conducted by a third party clinical research organisation (CRO: HCD Economics). Study findings will be published in peer-reviewed journals and related conferences.REC name
Wales REC 4
REC reference
19/WA/0226
Date of REC Opinion
6 Nov 2019
REC opinion
Further Information Favourable Opinion