ADVANCE-CFTR
Research type
Research Study
Full title
Advanced Diagnostic Validation and Novel Clinical Evaluation across the CFTR spectrum: a prospective study of CFTR functional assays, including rectal organoids, to improve the accuracy of diagnosing cystic fibrosis
IRAS ID
362887
Contact name
Nicholas Simmonds
Contact email
Sponsor organisation
National Heart and Lung institute, Royal Brompton Hospital
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Sometimes it is very difficult to tell if someone has cystic fibrosis (CF), especially when they have rare CF genes. Without this certainty, they are unlikely to get the correct treatment so their health will deteriorate. Some people wait for a long time, even decades, for their diagnosis to be confirmed, which causes huge distress for them and their families. More accurate ways to test for CF are therefore needed in this situation. Exciting new research using what are known as “mini-organs” shows great promise to solve this problem. These mini-organs, sometimes called organoids, are grown in laboratories using a small piece of gut tissue from a person. This is usually done through a quick, painless outpatients procedure. As they have the person’s exact genes, they can show if the CF gene is not working correctly. They can also help to work out the best treatment for that individual. We wish to carry out this research to prove that gut organoids are the best way to test for CF in this situation and then establish a UK organoid programme. This will help diagnose people faster and get the best treatment quicker, both key for leading a longer and healthier life.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
25/LO/0863
Date of REC Opinion
6 Jan 2026
REC opinion
Further Information Favourable Opinion