An observational study in Cystic Fibrosis, sponsored by Royal College of Surgeons, Ireland. Not yet recruiting. Open to participants aged 1 Month to 5 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2023-08-31.
Sponsored by Royal College of Surgeons, Ireland · Observational
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's planned enrollment of 550 is above the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Royal College of Surgeons, Ireland is the lead sponsor of 93 studies on the registry; 44 are open to participants now.
Counted across the registry records on this site, refreshed daily.
All participants with CF will be invited to participate in ENHANCE. We will ensure a presentative mix of mutation groups, sex, ages, ethnicity and location in all cohorts. We will target recruitment to cohorts 1 and 2 based on the following split of mutations: 50% F508del homozygous(FF), 20%heterozygous for F508del and a minimum function mutation(FMF), 20% heterozygous for F508del and a residual function/gating mutation or gating/other mutation and 10% with no currently treatable mutation(NON).
Children with CF attending one of the study centres and fulling one of the following:
Exclusion Criteria:
Newborn infants diagnosed with Cystic Fibrosis at newborn screening
Other: Quality of Life
Children with previous diagnosis of Cystic Fibrosis up to 5 years of age
Other: Quality of Life
Newborn infants without cystic fibrosis
Other: Quality of Life
ENHANCE will collect natural history on all children with cystic fibrosis who are enrolled over a 5 year period
1. The incidence, prevalence and progression of structural lung disease
Spirometry-controlled Computed Tomography
Time frame: 60 Months
2. The long-term natural history of pulmonary function and ventilation homogeneity.
Spirometry, Multiple Breath Washout
Time frame: 60 Months
3. The incidence, prevalence and longitudinal progression of CF liver disease.
Liver Ultrasound, Liver Function Tests
Time frame: 60 Months
4. The prevalence, natural history and progression of exocrine pancreatic dysfunction
Faecal Elastase Analysis
Time frame: 60 Months
5. The longitudinal natural history of gastrointestinal symptoms, inflammation and the gut microbiome compared to a healthy control population
Microbiome Analysis, Identification of inflammatory markers, Abdominal Symptom Scores
Time frame: 60 Months
6. The longitudinal natural history of annual sweat chloride levels in infants and children of different ages, the influence of different treatments on this and its association with other outcomes
Sweat chloride
Time frame: 60 Months
7. The longitudinal natural history of mental health outcomes in children with CF compared to controls.
Mental Health Quality Of Life Questionnaires
Time frame: 60 Months
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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Royal College of Surgeons, Ireland