Cystic FibrosisNCT03279965
MRI in Cystic Fibrosis and Primary Ciliary Dyskinesia
An observational study of Cystic Fibrosis and Primary Ciliary Dyskinesia, run by Royal Brompton & Harefield NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
This is a small pilot / feasibility study (Approximately 50 patients) to assess the possibility of clinical implementation of MRI assessment of patients with cystic fibrosis and primary ciliary dyskinesia. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis, Primary Ciliary Dyskinesia
- What is being tested?
- MRI
- How many people
- 50 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 5 May 2017to 5 May 2018
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with known cystic fibrosis or primary ciliary dyskinesia referred for CT chest as part of routine outpatient or inpatient investigations.
Who may be able to join
- Known CF or PCD Referred for CT chest
Who cannot join
- Contraindication to MRI (Pacemaker etc) Unable to stay still for MRI
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Royal Brompton Hospital
London, SW3 6NP
Thomas Semple, FRCR MBBS, 02073528121, T.Semple@rbht.nhs.uk
Anand Devaraj, 02073528121, A.Devaraj@rbht.nhs.uk
About this study
Official title: A Pilot Study to Assess the Use of MRI in the Assessment of Patients With Cystic Fibrosis and Primary Ciliary Dyskinesia
This is a small pilot / feasibility study (Approximately 50 patients) to assess the possibility of clinical implementation of MRI assessment of patients with cystic fibrosis and primary ciliary dyskinesia.
Patients will undergo their standard CT imaging and lung function investigations and additionally will undergo MRI examination. Reports from CT (the current gold standard) and MRI will be assessed for concordance and patient acceptability and examination implementation costs will also be assessed. Novel MRI-based potential markers of CF and PCD disease state will also be assessed.
Read the detailed description
In this small single site pilot / feasibility study we aim to recruit approximately 50 patients over the age of 6 years with known cystic fibrosis (CF) or primary ciliary dyskinesia (PCD).
Patients will be recruited from routine clinic appointments or at admission for inpatient investigation and/or treatment at the point of referral for computed tomography (CT) examination. If they consent to participation they will undergo MRI examination in addition to their standard clinical CT (within 7 days of the CT scan - on the same day if practicable).
The magnetic resonance imaging (MRI) scans will be anonymised and reported by radiologists, blinded to the CT findings. The reports and scores (Eichinger and Brody2) will then be compared those of the standard CT to assess concordance between the modalities and inter and intra observer variability.
Potential novel biomarkers (pulmonary ventilation and perfusion (non-contrast) and sinus mucus and liver tissue characteristics) will be compared with established markers (including lung clearance index) and known CF mutation / PCD type.
Patients or their carers will also be asked to fill in a short questionnaire comparing the differential patient acceptability of CT and MRI examinations. The cost (time, resources etc) of each examination will be calculated and compared.
- Conditions
- Cystic Fibrosis, Primary Ciliary Dyskinesia
- Being tested
- MRI
- Keywords
- Lung MRI cystic fibrosis primary ciliary dyskinesia
- Medical subject headings
- Cystic Fibrosis, Ciliary Motility Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIDiagnostic test
Also called Nuclear magnetic resonance (NMR)
MRI of lungs, paranasal sinuses and liver in addition to established clinical examinations (lung CT, pulmonary function testing)
| Study group | Role | What they receive |
|---|---|---|
| Cystic fibrosis | Patients with known cystic fibrosis Receives: Diagnostic Test: MRI | |
| Primary ciliary dyskinesia | Patients with known primary ciliary dyskinesia Receives: Diagnostic Test: MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
Structural and quantitative MRI vs CT in cystic fibrosis and primary ciliary dyskinesia assessment
To determine whether MRI with ventilation imaging can produce sufficiently diagnostic images, in the setting of CF or PCD, to replace CT imaging follow up. This will be assessed via semi-quantitative visual scoring (CT-based Brody / CFCT score and MRI-based Eichinger score) with both scoring systems applied to both CT and MRI. Scoring of the "air-trapping" component of CFCT will be substituted with scoring of ventilation defects on MRI. Scores will come from 2 observers, blinded to each others opinion, with a 3rd observer acting to resolve discrepancies. Intermodality variation will be assessed via ICC and Bland-Altman
Measured: 18 months
Secondary outcomes (8)
Ventilation MRI vs Lung clearance index (LCI)
Dynamic oxygen enhanced MRI measures will be compared to equivalent measurements from lung clearance testing. Bland Altman and ICC analysis of time to oxygen wash in (in seconds) on MRI, to time to nitrogen washout in seconds from lung clearance testing
Measured: 18 months
Sinus disease and exacerbations 1
Simple correlative statistics of degree of sinus opacification (percentage volume occupied by mucus and polyps) with frequency of infective exacerbations (number per month)
Measured: 18 months
Sinus disease and exacerbations 2
Simple correlative statistics of degree of sinus opacification (percentage volume occupied by mucus and polyps) with rate of decline in spirometry measures (percentage drop in predicted FEV1 and FEV1/FVC over the 6 months prior to scanning)
Measured: 18 months
Sinus disease and exacerbations 3
Simple correlative statistics of presence of susceptibility artefact in sinus mucus (binary; yes, no) with micro-organism cultured (nominal)
Measured: 18 months
Liver disease 1
Simple correlation of liver volume (cm3) and T1 relaxation time (ms) with serologic markers of liver function (AST, ALT and ALP in IU/L and albumin in g/L)
Measured: 18 months
Liver disease 2
Simple correlation of liver T1 relaxation time (ms) with ultrasound elastography markers of liver fibrosis (sheer wave speed in m/s)
Measured: 18 months
Patient acceptability
To determine whether MRI is as acceptable to patients with CF or PCD as CT. This is via simple descriptive statistics from a bespoke questionnaire consisting of 5-point Likert scale questions regarding each scan.
Measured: 18 months
Clinical implementation
To determine the operational cost differences in implementing MRI follow up vs CT follow up. Simple comparison of cost (in GBP) of diagnostic CT vs lung MRI.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2017
- Study start5 May 2017
- Primary completion5 May 2018 (estimated)
- Study completion5 May 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 only
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Royal Brompton & Harefield NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Bioxydyn Ltd, Manchester, University Hospital, Basel, Switzerland
- Responsible party
- Sponsor
- Investigators
- Anand Devaraj, Principal investigator, Royal Brompton Hospital, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03279965
- Sponsor's protocol ID
- 2016LI002B
- Status last verified
- Sep 2017
- First submitted
- 19 Jun 2017
- Last updated
- 12 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan to share individual participant data outside the research team at this time.
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Assess the Use of MRI in the Assessment of Patients With Cystic Fibrosis and Primary Ciliary Dyskinesia
- ClinicalTrials.gov ID
- NCT03279965
- Acronym / sponsor ID
- 2016LI002B
- Conditions
- Cystic Fibrosis, Primary Ciliary Dyskinesia
- Keywords
- Lung MRI cystic fibrosis primary ciliary dyskinesia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Royal Brompton & Harefield NHS Foundation TrustUniversity, hospital or other
- Collaborators
- Bioxydyn Ltd, Manchester, University Hospital, Basel, Switzerland
- Study start
- 5 May 2017 (actual)
- Primary completion
- 5 May 2018 (estimated)
- Study completion
- 5 May 2018 (estimated)
- First posted
- 12 Sep 2017 (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.
This is a small pilot / feasibility study (Approximately 50 patients) to assess the possibility of clinical implementation of MRI assessment of patients with cystic fibrosis and primary ciliary dyskinesia.
Patients will undergo their standard CT imaging and lung function investigations and additionally will undergo MRI examination. Reports from CT (the current gold standard) and MRI will be assessed for concordance and patient acceptability and examination implementation costs will also be assessed. Novel MRI-based potential markers of CF and PCD disease state will also be assessed.
Detailed description
In this small single site pilot / feasibility study we aim to recruit approximately 50 patients over the age of 6 years with known cystic fibrosis (CF) or primary ciliary dyskinesia (PCD).
Patients will be recruited from routine clinic appointments or at admission for inpatient investigation and/or treatment at the point of referral for computed tomography (CT) examination. If they consent to participation they will undergo MRI examination in addition to their standard clinical CT (within 7 days of the CT scan - on the same day if practicable).
The magnetic resonance imaging (MRI) scans will be anonymised and reported by radiologists, blinded to the CT findings. The reports and scores (Eichinger and Brody2) will then be compared those of the standard CT to assess concordance between the modalities and inter and intra observer variability.
Potential novel biomarkers (pulmonary ventilation and perfusion (non-contrast) and sinus mucus and liver tissue characteristics) will be compared with established markers (including lung clearance index) and known CF mutation / PCD type.
Patients or their carers will also be asked to fill in a short questionnaire comparing the differential patient acceptability of CT and MRI examinations. The cost (time, resources etc) of each examination will be calculated and compared.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cystic fibrosis | – | Diagnostic Test: MRIPatients with known cystic fibrosis |
| Primary ciliary dyskinesia | – | Diagnostic Test: MRIPatients with known primary ciliary dyskinesia |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with known cystic fibrosis or primary ciliary dyskinesia referred for CT chest as part of routine outpatient or inpatient investigations.
Key inclusion criteria (1 listed)
- Known CF or PCD Referred for CT chest
Key exclusion criteria (1 listed)
- Contraindication to MRI (Pacemaker etc) Unable to stay still for MRI
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRIAlso: Nuclear magnetic resonance (NMR)MRI of lungs, paranasal sinuses and liver in addition to established clinical examinations (lung CT, pulmonary function testing) | Diagnostic test | Cystic fibrosis, Primary ciliary dyskinesia |
Primary outcome
Structural and quantitative MRI vs CT in cystic fibrosis and primary ciliary dyskinesia assessment
Time frame: 18 months
To determine whether MRI with ventilation imaging can produce sufficiently diagnostic images, in the setting of CF or PCD, to replace CT imaging follow up. This will be assessed via semi-quantitative visual scoring (CT-based Brody / CFCT score and MRI-based Eichinger score) with both scoring systems applied to both CT and MRI. Scoring of the "air-trapping" component of CFCT will be substituted with scoring of ventilation defects on MRI. Scores will come from 2 observers, blinded to each others opinion, with a 3rd observer acting to resolve discrepancies. Intermodality variation will be assessed via ICC and Bland-Altman
8 secondary outcomes
Ventilation MRI vs Lung clearance index (LCI)
Time frame: 18 months
Sinus disease and exacerbations 1
Time frame: 18 months
Sinus disease and exacerbations 2
Time frame: 18 months
Sinus disease and exacerbations 3
Time frame: 18 months
Liver disease 1
Time frame: 18 months
Liver disease 2
Time frame: 18 months
Patient acceptability
Time frame: 18 months
Clinical implementation
Time frame: 18 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2017.
- Individual participant dataSharing planned: No. There is no plan to share individual participant data outside the research team at this time.
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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.
Anand Devaraj principal investigator
Royal Brompton Hospital, London
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 Primary Ciliary Dyskinesia, run by Royal Brompton & Harefield 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 1 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 12 Sep 2017. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anand Devaraj (02073528121, A.Devaraj@rbht.nhs.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Structural and quantitative MRI vs CT in cystic fibrosis and primary ciliary dyskinesia assessment (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Sep 2017; AzTrials last refreshed its copy on 7 Nov 2026.