Questionnaire review of Anderson-Fabry gastrointestinal symptoms
Research type
Research Study
Full title
Questionnaire based study of Anderson-Fabry patients disease related gastrointestinal symptoms compared to unaffected control group.
IRAS ID
177547
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
R&D at Royal Free Hospital
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
This study will involve posting questionnaires to patients with Anderson-Fabry disease (AFD) who currently receive treatment and follow up at the Royal Free Hospital Lysosomal Storage Disorders Unit. The questionnaire will be comprised of questions pertaining to patients experience of gastrointestinal symptoms relating to their disease, both before and after diagnosis and treatment. The patients will be requested to return the questionnaire pre-addressed envelopes to the Lysosomal Storage Disorders Unit at the Royal Free Hospital. The questionnaires will be distributed on a pseudoynomised basis, with no patients identifiable information on the questionnaire. A unique number will appear in the questionnaire and will correspond to the patient. This will allow for accurate information around date of diagnosis, time of commencing treatment, objective measures of gastrointestinal health (eg weight/BMI) to be elicited without relying on patient recall in order to to decrease areas of bias. An adapted version of the questionnaire will be distributed amongst a control group of healthy individuals for the purposes of comparison.
REC name
West of Scotland REC 5
REC reference
15/WS/0193
Date of REC Opinion
17 Sep 2015
REC opinion
Further Information Favourable Opinion