ENHANCE- Establishing Natural History in an Advanced New CF Care Era

Measured outcomes for people with CF have improved dramatically over the last 20 years, even prior to the widespread introduction of cystic fibrosis transmembrane conductance regulator (CTFR) modulator treatments. The outlook for children with CF has improved significantly, with longer predicted survival and a lower likelihood of morbidity. This has accelerated recently. These changes have occurred within a short period of time, and there is much that we now do not understand about disease progression in children with CF and how this differs from children without CF. CF is an area which is fortunate to have well-developed and successful disease registries. CF registries have provided significant amounts of very useful data to guide improvement in treatment and outcomes over many decades. The power of registries comes from the collection of a well-defined set of important outcome measures in very large numbers of people over many years. The outcome measures collected routinely in clinical care, which form part of the registries, are helpful in monitoring moderate-advances and symptomatic disease in people with CF. CF registries however do not tend to collect tomography(CT) scores, lung clearance index(LCI) or indeed repeated collection of biomarkers of disease activity such as sweat chloride which are increasingly relevant in an era of modulator therapies and reducing burden of symptomatic disease. We perceive an urgent need to complement registry data, cataloguing the changing natural history if early childhood CF by proactively collecting and curating sensitive, meaningful outcome data in a large cohort of children during this new era in Ireland and the UK. The prevalence, presentation and natural history of disease manifestation of CF in young children will change significantly in the next decade with advances in the understanding and treatment of CF, including the use of therapies aimed at CFTR function. ENHANCE provides an opportunity to study these changes in real-time and in ways that are relevant to the CF community.

NCT Number	NCT05986045
Lead Sponsor	Royal College of Surgeons, Ireland Collaborators: University Hospital of Limerick, Cork University Hospital, University College Hospital Galway, Belfast Health and Social Care Trust, NHS Lothian, Alder Hey Children's NHS Foundation Trust, Manchester University NHS Foundation Trust, Newcastle-upon-Tyne Hospitals NHS Trust, Cardiff and Vale University Health Board, Royal Brompton & Harefield NHS Foundation Trust, Erasmus University Rotterdam, Medizinische Hochschule Brandenburg Theodor Fontane, Massachusetts General Hospital, The Hospital for Sick Children, Teagasc
Conditions	Cystic Fibrosis
Enrollment	550 participants
Start Date	2023-10
Primary Completion	2028-09-30 (estimated)
Study Completion	2028-09-30 (estimated)
Updated on ClinicalTrials.gov	2023-08-31