Gut Imaging for Function & Transit in Cystic Fibrosis Study 2

• Status: Completed
• Trial end date: 2020-10-29
• Target enrollment: 0
• Participant gender: All

Summary

People with Cystic Fibrosis (CF) have problems digesting their food properly. More than 8 in 10 people with CF must take medication to assist their digestion. In spite of this, complications such as bowel blockage occur.

Finding out how already licenced drugs for CF work in the gut is the first step in repurposing medications. Tezacaftor/Ivacaftor with Ivacaftor is a drug combination which corrects the basic defect in CF an has shown improvements on lung function.

The purpose of this study is to evaluate, using Magnetic Resonance Imaging (MRI) and patient-reported outcomes, whether Tezacaftor/Ivacaftor with Ivacaftor has an effect on improving gastrointestinal problems in CF.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Nottingham University Hospitals NHS Trust
• Collaborators: Cystic Fibrosis Foundation; Cystic Fibrosis Trust; Nottingham University Hospitals Charity; University of Nottingham; Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Inclusion Criteria:

• capacity to consent, or to understand the requirements of the study where parental consent is needed

• confirmed diagnosis of CF, either by sweat test or genetic testing. Tezacaftor/Ivacaftor is indicated only for patients homozygous for the commonest CF mutation - p.Phe508del and so we will enrol only CF patients with this genotype

Exclusion Criteria:

• currently taking CFTR modulator drug

• Contra-indication to use of Tezacaftor/Ivacaftor

• Measurement of FEV1 <40% predicted using Global Lung Initiative criteria, according to clinical records, as participants will be required to perform a series of 10 second breathholds throughout MRI scanning

• Contra-indication to MRI scanning, such as embedded metal, pacemaker.

• Pregnancy

• Unable to stop medications directly prescribed to alter bowel habit, such as laxatives or anti-diarrhoeals, on the study day

• Previous resection of any part of the gastrointestinal tract apart from appendicectomy or cholecystectomy. Surgical relief of meconium ileus or DIOS will be permitted unless clinical records show excision of intestine >20cm in length.

• Intestinal stoma

• Diagnosis of inflammatory bowel disease or coeliac disease confirmed by biopsy

• Gastrointestinal malignancy

• Unable to comply with dietary restrictions required for the study.