Cystic Fibrosis Clinical Trial
— CFTR-MAGICOfficial title:
CFTR Modulators and Gastrointestinal Complications
Verified date | July 2022 |
Source | Nottingham University Hospitals NHS Trust |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Active, not recruiting |
Enrollment | 40000 |
Est. completion date | September 30, 2023 |
Est. primary completion date | September 30, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: - All patients with CF of any genotype on the UK and US cystic fibrosis registries from the period 2007 - 2018. Exclusion Criteria: - Patients whose CFTR modulator status is unknown or only have one year of CFTR data recorded on the registry will be excluded from the analysis of the effects of CFTR modulators. This is to account for the fact that DIOS data is annualised on the registries, therefore there is no certainty at what time of year DIOS was diagnosed in relation to commencing CFTR modulator therapy. |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Nottingham University Hospitals NHS Trust | Nottingham |
Lead Sponsor | Collaborator |
---|---|
Nottingham University Hospitals NHS Trust | Royal Brompton & Harefield NHS Foundation Trust, Seattle Children's Hospital, The Leeds Teaching Hospitals NHS Trust, University of Washington |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Describe DIOS events and PERT usage in UK and US registries 2007-2018 | Descriptive analysis to include cumulative incidence of DIOS and prevalence of PERT usage in the UK and US. PERT use will be dichotomised (i.e use/no use), incidence of DIOS (event/no event), plotted by year, separated by registry. Trends will be described according to age range (<6, 6-11, 12+), sex, BMI, ethnicity, antibiotic use, gastrointestinal co-morbidities and FEV1 lung function.
These demographics will also be used for demographic propensity matching to define a control group of non-eligible non-users of CFTR modulators that are otherwise representative of the CFTR modulator user group the year that the CFTR modulator exposure was commenced. |
2 years | |
Primary | Determine the effect of CFTR modulators on the incidence of DIOS and use of PERT: population level controlled interrupted time series (ITS) | This will investigate the effect of CFTR modulator intervention on the rate on PERT use and DIOS incidence.
ITS will explore proportion of DIOS events at population level in the CFTR modulator population and the propensity score defined comparator population. ITS will explore proportion of overall PERT usage (user/non user), and the proportion of individuals with a reduction in weight standardised PERT dose (decrease/no decrease). |
2 years | |
Primary | Determine the effect of CFTR modulators on the incidence of DIOS and use of PERT: patient-level time series | This will focus on patient level associations to complement the registry level findings. Associations in the incidence of PERT usage and DIOS events from 2012 in individuals taking CFTR modulators, compared to propensity matched controls will be explored.
DIOS events: Multilevel Poisson Regression model for count data by year, incidence rate ratio (frequency of DIOS events). Adjusted analysis for main covariates. PERT use: Multilevel linear regression for continuous data, multilevel logistic regression for dichotomised data. Adjusted for main covariates. |
2 years |
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