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

NCT ID: NCT04277819

Last Updated: 2024-05-01

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 157 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-02-15
• Study Completion Date: 2023-06-21

Brief 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.

Conditions

Cystic Fibrosis; Liver Fibroses; Cystic Fibrosis Liver Disease

Study Design

• Observational Model Type: CASE_CONTROL
• Study Time Perspective: CROSS_SECTIONAL

Study Groups

Patients with cystic fibrosis related liver disease

Patients with cystic fibrosis, who meet the criteria for diagnosis of liver disease according to the European Cystic Fibrosis Society best practice guidelines

No interventions assigned to this group

Patients without cystic fibrosis related liver disease

No interventions assigned to this group

Eligibility Criteria

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 β-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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Manchester University NHS Foundation Trust

OTHER_GOV

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Wythenshawe Hospital

Manchester, , United Kingdom

Countries

United Kingdom

References

Scott JA, Jones AM, Jokl E, Gordon-Walker T, Barry PJ, Hanley NA, Piper Hanley K, Athwal VS. Improving detection of cystic fibrosis related liver disease using liver fibrosis assessment tools. Heliyon. 2023 Nov 7;9(11):e21861. doi: 10.1016/j.heliyon.2023.e21861. eCollection 2023 Nov.

Reference Type: DERIVED

PMID: 38053913 (View on PubMed)

Other Identifiers

B00306

Identifier Type: -

Identifier Source: org_study_id