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

Summary

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.

Conditions

• Cystic Fibrosis

Interventions

OTHER

Quality of Life

ENHANCE will collect natural history on all children with cystic fibrosis who are enrolled over a 5 year period

Sponsors & Collaborators

• University Hospital of Limerick

  collaborator OTHER

• Cork University Hospital

  collaborator OTHER

• University College Hospital Galway

  collaborator OTHER

• Belfast Health and Social Care Trust

  collaborator OTHER

• NHS Lothian

  collaborator OTHER_GOV

• Alder Hey Children's NHS Foundation Trust

  collaborator OTHER

• Manchester University NHS Foundation Trust

  collaborator OTHER_GOV

• Newcastle-upon-Tyne Hospitals NHS Trust

  collaborator OTHER

• Cardiff and Vale University Health Board

  collaborator OTHER_GOV

• Royal Brompton & Harefield NHS Foundation Trust

  collaborator OTHER

• Erasmus University Rotterdam

  collaborator OTHER

• Medizinische Hochschule Brandenburg Theodor Fontane

  collaborator OTHER

• Massachusetts General Hospital

  collaborator OTHER

• The Hospital for Sick Children

  collaborator OTHER

• Teagasc

  collaborator INDUSTRY

• Royal College of Surgeons, Ireland

  lead OTHER

Principal Investigators

• Paul McNally · RCSI

Eligibility

Min Age

1 Month

Max Age

5 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2023-10-31

Primary Completion

2028-09-30

Completion

2028-09-30