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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04277819
• Other study ID #: B00306
• Status: Completed
• Start date: February 15, 2019
• Est. completion date: June 21, 2023

Study information

• Verified date: April 2024
• Source: Manchester University NHS Foundation Trust
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 157
• Est. completion date: June 21, 2023
• Est. primary completion date: June 21, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Male or female > 18 years of age

2. Females will be non-pregnant and non-lactating* (for MRI scan only)

3. 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:

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

Exclusion Criteria:

1. Contraindication to magnetic resonance imaging scanning (including claustrophobia) or gadolinium-based contrast agent

2. eGFR < 50 mL/min/1.73m2

3. Pregnant or breast-feeding women.

4. Any other condition, which in the opinion of the research team may put participants at risk during the study.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Cystic Fibrosis Liver Disease
• Fibrosis
• Liver Cirrhosis
• Liver Diseases
• Liver Fibroses

Locations

Country	Name	City	State
United Kingdom	Wythenshawe Hospital	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Manchester University NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Diagnostic criteria of CFLD	Number of participants that would be diagnosed with cystic fibrosis related liver disease according to the current European CF guidelines.	2 years
Primary	Using FibroScan as a tool to increase detection of CLFD	Number of participants that would be diagnosed with cystic fibrosis related liver disease with the addition of FibroScan to the diagnostic criteria	2 years
Primary	Using Biomarkers as a tool to detect CFLD	Number of participants with a diagnosis of CFLD that have an increase in serum biomarker values for known biomarkers of liver fibrosis.	2 years
Primary	Using MRI scan as a tool to detect CFLD	Number of participants with a diagnosis of CFLD that show an increase in fibrosis on their MRI scan.	2 years