The Use of Novel Diagnostic Tools to Increase Detection of Early Fibrosis in Cystic Fibrosis Related Liver Disease to Improve Clinical Management

Cystic Fibrosis (CF) is a genetic condition which affects 1 in 2500 newborn infants and is the commonest genetic condition in the UK. 1 in 25 of the white population carry the mutation. The genetic defect prevents the movement of fluids from cells, leading to thickened secretions and injury. With improvements in treatments from the commonest organ affected, the lungs, patients born with CF now can expect to live into their 40s with more than 60% living past 16.

Though better, more can be done. As treatments from lung complications have improved, the management of liver disease (second commonest organ involved) remains unchanged for a considerable time. Treatment options are limited with liver transplant the only curative option. Though potentially life-saving, it has risks and an organ shortage means alternative treatment options are desperately needed.

Identifying those with or at risk of Cystic Fibrosis related liver disease is difficult due to inadequate diagnostic tools. Routine blood tests are unreliable; therefore specific blood tests to identify scarring of the liver (biomarkers) are urgently needed. Ultrasound scan, the recommended diagnostic investigation, is only accurate in identifying the late stages of liver disease. For new therapies to be most effective we need to be able to identify patients at a much earlier stage.

This study will use multi-modality testing, including imaging techniques such as FibroScan, MRI scan and blood tests (biomarkers), to diagnose those with liver scarring and use this to better categorise disease.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Liver Disease Digestive System Diseases Fibrosis Genetic Diseases, Inborn Infant, Newborn, Diseases Liver Cirrhosis Liver Diseases Liver Fibroses Lung Diseases Pancreatic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Wythenshawe Hospital

Manchester, M23 9LT, United Kingdom

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Male or female > 18 years of age
Females will be non-pregnant and non-lactating* (for MRI scan only)
20 patients with confirmed diagnosis of CF, 20 with CFLD and 20 healthy volunteers

Women of childbearing potential (i.e. not surgically sterilised or <1 year post menopause) will be required to:

Confirm they are not currently breastfeeding 2. Undergo a serum pregnancy test (serum β-HCG)

Exclusion criteria

Contraindication to magnetic resonance imaging scanning (including claustrophobia) or gadolinium-based contrast agent
eGFR < 50 mL/min/1.73m2
Pregnant or breast-feeding women.
Any other condition, which in the opinion of the research team may put participants at risk during the study.

Treatment and study plan

Primary outcomes

Diagnostic criteria of CFLD

Time frame: 2 years

Number of participants that would be diagnosed with cystic fibrosis related liver disease according to the current European CF guidelines.
Using FibroScan as a tool to increase detection of CLFD

Time frame: 2 years

Number of participants that would be diagnosed with cystic fibrosis related liver disease with the addition of FibroScan to the diagnostic criteria
Using Biomarkers as a tool to detect CFLD

Time frame: 2 years

Number of participants with a diagnosis of CFLD that have an increase in serum biomarker values for known biomarkers of liver fibrosis.
Using MRI scan as a tool to detect CFLD

Time frame: 2 years

Number of participants with a diagnosis of CFLD that show an increase in fibrosis on their MRI scan.

Sponsors and collaborators

Lead sponsor

Manchester University NHS Foundation Trust

Other Gov

Registry information

Important dates

Study start: 2019
Primary completion: 2023
Study completion: 2023
First posted: Feb 20, 2020
Registry last updated: May 1, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed