ENHANCE (Establishing Natural History in an Advanced New CF Care Era)
Research type
Research Study
Full title
Establishing Natural History in an Advanced New CF Care Era (ENHANCE)
IRAS ID
332109
Contact name
Jane Davies
Contact email
Sponsor organisation
Royal College of Surgeons in Ireland
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
The introduction of a new generation of CF medicines has results in remarkable improvements in health for many. Use of one such treatment, Ivacaftor, has been associated with outcomes which, in many cases, are similar to health children. As more effective treatments are introduced at earlier ages, the paediatric CF community will need to move away from measuring improvements in existing disease to figuring out whether disease can be prevents from occurring in the first place. Registries have been really helpful for people with CF, collecting lots of useful patient information over years and helping us to understand how CF develops and affects people. However, registries best measure signs of more advanced disease such as what is commonly seen in adults. If we want to really understand what is happening in children in this new age of CF, as well as starting from birth and following more children for many years, we need much more sensitive tests that detect early and mild disease better.
In this first 5 years of ENHANCE, working with parents and people with CF and building on our success with the RECOVER study, we will recruit up to 450 young children with CF and 100 healthy controlled across 13 study sites in Ireland and the UK. We will collect information just once per year so that the study doesn’t overly interfere with lives and gives us the best chance to get a clear picture of how CF affects the children as they grow. We will use the most sensitive tests to detect the earliest signs of lung disease. We will use powerful research tools to understand the nature of abdominal complaints, CF liver disease, pancreatic status and mental health in children with CF in this new era. Through ENHANCE we will develop a clearer understanding of the impact of treatments in young children with CF, and what this might mean for the real questions, like how we can enable children with CF to lead a long, fulfilling life, not defined by disease.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0921
Date of REC Opinion
4 Jan 2024
REC opinion
Further Information Favourable Opinion