Cystic FibrosisNCT04277819
The Use of Novel Diagnostic Tools to Increase Detection of Early Fibrosis in Cystic Fibrosis Related Liver Disease to Improve Clinical Management
An observational study of Cystic Fibrosis and Liver Fibroses, run by Manchester University NHS Foundation Trust at 1 site in United Kingdom.
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Liver Fibroses, Cystic Fibrosis Liver Disease
- How many people
- 157 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 15 Feb 2019to 21 Jun 2023
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: The study population will be selected from the Manchester Adult Cystic Fibrosis Centre. For the initial stage of the study 200 patients will be selected who are attending for their annual reviews. At this stage they will be consented, have bloods taken and FibroScan performed. From this initial cohort, they will be classified into presence and absence of liver disease based on current guidelines. From this 20 patients from each group along with 20 healthy volunteers will then undergo MRI scan.
Who may be able to join
- 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:
- 1. Confirm they are not currently breastfeeding 2. Undergo a serum pregnancy test (serum β-HCG)
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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.
- Medical subject headings
- Cystic Fibrosis, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 | |
| Patients without cystic fibrosis related liver disease | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Diagnostic criteria of CFLD
Number of participants that would be diagnosed with cystic fibrosis related liver disease according to the current European CF guidelines.
Measured: 2 years
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
Measured: 2 years
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.
Measured: 2 years
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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2020
- Study start15 Feb 2019
- Primary completion21 Jun 2023
- Study completion21 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 157 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Manchester University NHS Foundation Trust
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04277819
- Sponsor's protocol ID
- B00306
- Status last verified
- Apr 2024
- First submitted
- 11 Feb 2020
- Last updated
- 1 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 38053913
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Use of Novel Diagnostic Tools to Increase Detection of Early Fibrosis in Cystic Fibrosis Related Liver Disease to Improve Clinical Management
- ClinicalTrials.gov ID
- NCT04277819
- Acronym / sponsor ID
- B00306
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 157 participants (actual)
- Lead sponsor
- Manchester University NHS Foundation TrustOther government
- Study start
- 15 Feb 2019 (actual)
- Primary completion
- 21 Jun 2023 (actual)
- Study completion
- 21 Jun 2023 (actual)
- First posted
- 20 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Target follow-up
- 2 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
| Patients without cystic fibrosis related liver disease | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- The study population will be selected from the Manchester Adult Cystic Fibrosis Centre. For the initial stage of the study 200 patients will be selected who are attending for their annual reviews. At this stage they will be consented, have bloods taken and FibroScan performed. From this initial cohort, they will be classified into presence and absence of liver disease based on current guidelines. From this 20 patients from each group along with 20 healthy volunteers will then undergo MRI scan.
Key inclusion criteria (5 listed)
- 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
- 1. Confirm they are not currently breastfeeding 2. Undergo a serum pregnancy test (serum β-HCG)
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2024.
- Derived from this study: PubMed 38053913Scott 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis and Liver Fibroses, run by Manchester University NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Diagnostic criteria of CFLD (time frame: 2 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04277819, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2024.