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

Sponsor
Royal College of Surgeons, Ireland
Study ID
NCT05986045
Status
Not Yet Recruiting

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Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
1 Month - 5 Years
Healthy Volunteers
Accepted

Interventions

  • Quality of Life — OTHER
    ENHANCE will collect natural history on all children with cystic fibrosis who are enrolled over a 5 year period

Study Details

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.

Key Dates

First listed
Aug 14, 2023
Start date
Oct 31, 2023
Status verified
Jul 2023
Primary completion
Sep 30, 2028
Completion
Sep 30, 2028

Study Design

Enrollment
550 participants (estimated)

Arms

  • Arm: Cohort 1
    Newborn infants diagnosed with Cystic Fibrosis at newborn screening
  • Arm: Cohort 2
    Children with previous diagnosis of Cystic Fibrosis up to 5 years of age
  • Arm: Control
    Newborn infants without cystic fibrosis

Primary Outcome Measure

1. The incidence, prevalence and progression of structural lung disease [ Time Frame: 60 Months ]

Central Contacts

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