NCT05253859

CFTR Modulators and Gastrointestinal Complications

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 47,023 (actual)
• Sponsor: Nottingham University Hospitals NHS Trust · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

To elucidate the similarities and distinctions in non-pulmonary manifestations of cystic fibrosis (CF) including distal intestinal obstruction syndrome (DIOS) incidence and pancreatic enzyme replacement therapy (PERT) use between US and UK CF populations in a parallel study using data from the UK and US CF registries. To assess how CFTR modulators impacted upon recorded PERT use and incidence of DIOS.

Detailed description

Cystic fibrosis (CF) is a autosomal recessive multi-system disorder caused by mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) gene. As well as the documented respiratory complications of CF, gastrointestinal manifestations are of clinical importance. CFTR mutations in the gastrointestinal tract are responsible for pancreatic exocrine insufficiency in around 85% of people with CF (pwCF). In addition a severe gastrointestinal complication in CF is distal intestinal obstructive syndrome (DIOS), affecting 5.7% pwCF in the UK (2.5% in \<16 years and 7.7% in adults) and 2.1% in the US (\<18 years 1.7%, adult 2.4%) in 2019. This is a parallel data registry study using data from the UK and US CF registries, with data provided for the time period 2007-2018. As such no individual participants will be recruited to the study. The CFTR modulators to be studies are Ivacaftor and lumacaftor/ivacaftor. Study aims; * Describe DIOS events and PERT usage in UK and US registries * Determine the effect of CFTR modulators on the incidence of DIOS and use of PERT: population time series * Determine the effect of CFTR modulators on the incidence of DIOS and use of PERT: patient-level time series The outcomes of the above aims will be used to generate hypotheses regarding the effect of the newer CFTR modulators such as Symdeko/Symkevi and Tricaftor/Kaftrio on PERT usage and DIOS incidence in CF registry data post 2018. This will form the basis of future studies.

Conditions

• Cystic Fibrosis
• Gastrointestinal System Disease
• Distal Intestinal Obstruction Syndrome
• Pancreatic Enzyme Abnormality
• Pancreatic Disease
• Lung Diseases
• Digestive System Disease

Interventions

Type	Name	Description
OTHER	No intervention	Registry study: No intervention

Timeline

• Start date: 2021-10-01
• Primary completion: 2024-02-10
• Completion: 2024-02-10
• First posted: 2022-02-24
• Last updated: 2024-07-03

Locations

1 site across 1 country: United Kingdom