MRI in Cystic Fibrosis and Primary Ciliary Dyskinesia

Cystic Fibrosis Clinical Trial

Official title:

A Pilot Study to Assess the Use of MRI in the Assessment of Patients With Cystic Fibrosis and Primary Ciliary Dyskinesia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03279965
• Other study ID #: 2016LI002B
• Status: Recruiting
• Phase: N/A
• First received: June 19, 2017
• Last updated: September 11, 2017
• Start date: May 5, 2017
• Est. completion date: May 5, 2018

Study information

• Verified date: September 2017
• Source: Royal Brompton & Harefield NHS Foundation Trust
• Contact: Anand Devaraj
• Phone: 02073528121
• Email: A.Devaraj[@]rbht.nhs.uk
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: May 5, 2018
• Est. primary completion date: May 5, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

• Known CF or PCD Referred for CT chest

Exclusion Criteria:

• Contraindication to MRI (Pacemaker etc) Unable to stay still for MRI

Related Conditions & MeSH terms

• Ciliary Motility Disorders
• Cystic Fibrosis
• Dyskinesias
• Fibrosis
• Kartagener Syndrome
• Primary Ciliary Dyskinesia

Intervention

Diagnostic Test:
• MRI
MRI of lungs, paranasal sinuses and liver in addition to established clinical examinations (lung CT, pulmonary function testing)

Locations

Country	Name	City	State
United Kingdom	Royal Brompton Hospital	London

Sponsors (3)

Lead Sponsor	Collaborator
Royal Brompton & Harefield NHS Foundation Trust	Bioxydyn Ltd, Manchester, University Hospital, Basel, Switzerland

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	18 months
Secondary	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	18 months
Secondary	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)	18 months
Secondary	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)	18 months
Secondary	Sinus disease and exacerbations 3	Simple correlative statistics of presence of susceptibility artefact in sinus mucus (binary; yes, no) with micro-organism cultured (nominal)	18 months
Secondary	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)	18 months
Secondary	Liver disease 2	Simple correlation of liver T1 relaxation time (ms) with ultrasound elastography markers of liver fibrosis (sheer wave speed in m/s)	18 months
Secondary	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.	18 months
Secondary	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.	18 months