Cystic FibrosisNCT03320382
Multiple Breath Washout, a Clinimetric Dataset
An observational study of Cystic Fibrosis and Primary Ciliary Dyskinesia, run by Imperial College London at 1 site in United Kingdom.
Trial summary
Monitoring patients with chronic, inflammatory airways disease particularly in the early stages is hampered by the relative insensitivity of current outcome measures to detect subtle changes. Read more about this study
- Who is it for?
- Ages 2 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Primary Ciliary Dyskinesia, Bronchiectasis
- What is being tested?
- Multiple breath washout testing
- Type of treatment
- Medical device
- How many people
- 530 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 5 May 2017to Mar 2024
- 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 2 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients aged two and upwards with chronic airways disease including CF, PCD, Asthma and Bronchiectasis, Persistent bacterial bronchitis and sleep disordered breathing and obesity as well as aged matched healthy control patients will be approached to take part in the study. Healthy volunteers will only complete MBW and Spirometry for comparison with respiratory disease patients.
Who may be able to join
- For patients with the following respiratory disease, diagnosis confirmed by:
- CF: diagnosed by standard criteria
- PCD: diagnosed by ciliary beat frequency measurement, ciliary beat pattern analysis or electron microscopy of ciliary ultrastructure, or genetics
- Non-CF bronchiectasis: CT diagnosis of bronchiectasis and not fulfilling diagnostic criteria for CF or PCD
- Asthma: as diagnosed by standard diagnostic criteria of British Thoracic Society/Scottish Intercollegiate Guideline Network (BTS/SIGN) guidelines.
- Persistent bacterial bronchitis defined as a wet cough present for >1 month, usually with bronchoscopic evidence of chronic infection, that resolves with appropriate antibiotic therapy
Show all 9Show fewer
- Sleep Disordered Breathing.
- For healthy volunteers, these will be colleagues and staff contacts (including children) at a participating centre i.e. Royal Brompton Hospital, or will be siblings of patients.
- Written informed consent (assent from children of appropriate age) obtained.
Who cannot join
- Positive culture (within the last year of / receiving treatment for Mycobaterium tuberculosis or abscessus (due to cross-infection concerns).
- Pregnant or breastfeeding.
- Inability to understand or cooperate with the test(s).
- Inability to give informed consent, or withdrawal of informed consent.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Royal Brompton Hospital
London, sw36lr
Katie J Bayfield, 02075947949, k.bayfield@imperial.ac.uk
Clare Saunders, 02075947949, c.saunders@imperial.ac.uk
About this study
Official title: Multiple Breath Washout in Paediatric Chronic Airways Disease: Building a Clinimetrics Dataset
Monitoring patients with chronic, inflammatory airways disease particularly in the early stages is hampered by the relative insensitivity of current outcome measures to detect subtle changes. Multiple breath washout is a potential sensitive test that is a useful readout of disease at these early stages but it lacks standardisation and knowledge of variability with reference to standard lung function measures. This is a Cross sectional and longitudinal observation study. The hypothesis is that multiple breath washout-derived indices will provide a robust signal of gas mixing inhomogeneity, correlating with conventional measures of airway disease severity. Multiple breath washout performed on different devices will generate indices which correlate but differ in value.
Read the detailed description
Lung Clearance Index (LCI) has been shown to be more sensitive and perhaps more applicable in early stages of lung disease when compared to currently utilised physiological outcome measures, and yet it is not approved by regulatory agencies as a recognised surrogate outcome measure. This may highlight some of the lack of understanding within MBW; what is the best equipment to use, what is the minimal value of change to show an improvement in LCI, what is LCI's correlation with clinical outcome measures and what does the progression of LCI tell us about lung disease. These questions will be addressed with a view to contributing a body of data to guide decisions around its utility in both interventional drug trials and for monitoring in a clinical setting.
The aim of this study is to assess the suitability of LCI as a clinical outcome measure for both clinical trials and clinical monitoring of patients with inflammatory respiratory disease across the disease spectrum. Different testing modalities will be compared as well as assess the repeatability and validity of LCI for its determination as an outcome measure. Long-term follow up will allow an assessment of the relationship between LCI and future outcomes such as rate of decline in lung function and frequency of exacerbations, both recognised as relevant by regulatory agencies.
- Conditions
- Cystic Fibrosis, Primary Ciliary Dyskinesia, Bronchiectasis, Asthma, Bronchitis, Sleep Disorder
- Being tested
- Multiple breath washout testing
- Medical subject headings
- Cystic Fibrosis, Ciliary Motility Disorders, Bronchiectasis, Asthma, Bronchitis, Sleep Wake Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Multiple breath washout testingDevice
Also called Exhlayzer D (Ecomedics), Modified Innocor (Innovision), Amis 2000 (Respiratory Mass Spectrometer) (Innovision), EasyOne Pro (New Diagnostic Design (NDD) Medical Technologies Inc
Multiple breath washout testing will be completed on different devices over time
What is being measured
The primary outcome is the main question the study is designed to answer.
Lung Clearance Index
Multiple breath washout involves recording concentrations of a tracer gas whilst it is cleared from the lungs during normal tidal breathing. The tracer gas can either be an inert gas such as sulphur hexafluoride (SF6) that is washed out by room air, or resident Nitrogen (N2) that is cleared by 100% oxygen (O2). All tracer gases are traditionally cleared to 1/40th (or 2.5%) of their starting concentration. This procedure is performed in triplicate, each test will take around 5-10 minutes with wait time in-between.
Measured: Each testing session may take up to 90 minutes but most likely it will be under an hour. The MBW test will be completed at each visit and the LCI and other MBW results are obtained from data analysis post completion.
Secondary outcomes (3)
Spirometry
Spirometry is an effort dependant test designed to assess lung volumes.Spirometry will be performed according to ERS guidelines on a spirometer such as the Easyone with a disposable mouthpiece and filter. Three good quality measurements will be made and the best recorded in absolute values. Spirometry will only be collected in subjects age 6 and upwards (or if performed adequately at clinic visits at a younger age) since it is difficult to perform and produce reliable spirometry results in this young age
Measured: Spirometry will be completed at each visit (unless already taken at a clinical visit). Maximum 10 visits over 3 years
Disease specific Quality of life questionnaire
Quality of Life Questionnaires provide patient-reported outcomes and how subjects are feeling in relation to new treatments or generally with regard to their lung disease. For certain diseases such as cystic fibrosis, these outcome measures are emerging as clinical tools, so it will not be duplicated if it has already been performed. A questionnaire will be completed either on paper or a computer at each visit to compare physiological data with how a patient is feeling.
Validated Revised Cystic Fibrosis Questionnaire (CFQR) for CF patients. For Asthma patients, Paediatric Asthma Quality of life Questionnaire, Paediatric Asthma Caregivers Quality of Life Questionnaire, Asthma Control Test Score, Childhood Asthma Control Test score, Test for Respiratory and Asthma Control in Kids.
For Primary Cilary Dyskinesia the new validated PCD questionnaire. Healthy volunteers will not complete a questionnaire.
Measured: The quality of life questionnaire will be completed at each visit and compared to the primary outcome at each visit. Maximum 10 visits over 3 years
Review of clinically indicated tests
For patients with respiratory disease, some results taken for clinically indicated reasons either on the same day or close to the research day may be reviewed from clinical notes or hospital electronic data capture systems as part of this research for comparisons. These may include blood sample results, sputum cultures and clinically assessment from the multidisciplinary team within clinic letters.
Measured: Post each visit review of clinically indicated tests will take place and compared to the primary outcome. Maximum 10 visits over 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2017
- Study start5 May 2017
- Primary completionMar 2024 (estimated)
- Study completionMar 2024 (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.
- More trials run by Imperial College LondonThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 530 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Imperial College London
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jane C Davies, Professor, Principal investigator, Imperial College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03320382
- Sponsor's protocol ID
- 17/NI/0046
- Status last verified
- May 2022
- First submitted
- 17 May 2017
- Last updated
- 6 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multiple Breath Washout in Paediatric Chronic Airways Disease: Building a Clinimetrics Dataset
- ClinicalTrials.gov ID
- NCT03320382
- Acronym / sponsor ID
- 17/NI/0046
- Conditions
- Cystic Fibrosis, Primary Ciliary Dyskinesia, Bronchiectasis, Asthma, Bronchitis, Sleep Disorder
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 530 participants (estimated)
- Lead sponsor
- Imperial College LondonUniversity, hospital or other
- Study start
- 5 May 2017 (actual)
- Primary completion
- Mar 2024 (estimated)
- Study completion
- Mar 2024 (estimated)
- First posted
- 25 Oct 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.
Monitoring patients with chronic, inflammatory airways disease particularly in the early stages is hampered by the relative insensitivity of current outcome measures to detect subtle changes. Multiple breath washout is a potential sensitive test that is a useful readout of disease at these early stages but it lacks standardisation and knowledge of variability with reference to standard lung function measures. This is a Cross sectional and longitudinal observation study. The hypothesis is that multiple breath washout-derived indices will provide a robust signal of gas mixing inhomogeneity, correlating with conventional measures of airway disease severity. Multiple breath washout performed on different devices will generate indices which correlate but differ in value.
Detailed description
Lung Clearance Index (LCI) has been shown to be more sensitive and perhaps more applicable in early stages of lung disease when compared to currently utilised physiological outcome measures, and yet it is not approved by regulatory agencies as a recognised surrogate outcome measure. This may highlight some of the lack of understanding within MBW; what is the best equipment to use, what is the minimal value of change to show an improvement in LCI, what is LCI's correlation with clinical outcome measures and what does the progression of LCI tell us about lung disease. These questions will be addressed with a view to contributing a body of data to guide decisions around its utility in both interventional drug trials and for monitoring in a clinical setting.
The aim of this study is to assess the suitability of LCI as a clinical outcome measure for both clinical trials and clinical monitoring of patients with inflammatory respiratory disease across the disease spectrum. Different testing modalities will be compared as well as assess the repeatability and validity of LCI for its determination as an outcome measure. Long-term follow up will allow an assessment of the relationship between LCI and future outcomes such as rate of decline in lung function and frequency of exacerbations, both recognised as relevant by regulatory agencies.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients aged two and upwards with chronic airways disease including CF, PCD, Asthma and Bronchiectasis, Persistent bacterial bronchitis and sleep disordered breathing and obesity as well as aged matched healthy control patients will be approached to take part in the study. Healthy volunteers will only complete MBW and Spirometry for comparison with respiratory disease patients.
Key inclusion criteria (9 listed)
- For patients with the following respiratory disease, diagnosis confirmed by:
- CF: diagnosed by standard criteria
- PCD: diagnosed by ciliary beat frequency measurement, ciliary beat pattern analysis or electron microscopy of ciliary ultrastructure, or genetics
- Non-CF bronchiectasis: CT diagnosis of bronchiectasis and not fulfilling diagnostic criteria for CF or PCD
- Asthma: as diagnosed by standard diagnostic criteria of British Thoracic Society/Scottish Intercollegiate Guideline Network (BTS/SIGN) guidelines.
Key exclusion criteria (4 listed)
- Positive culture (within the last year of / receiving treatment for Mycobaterium tuberculosis or abscessus (due to cross-infection concerns).
- Pregnant or breastfeeding.
- Inability to understand or cooperate with the test(s).
- Inability to give informed consent, or withdrawal of informed consent.
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 |
|---|---|---|
| Multiple breath washout testingAlso: Exhlayzer D (Ecomedics), Modified Innocor (Innovision), Amis 2000 (Respiratory Mass Spectrometer) (Innovision), EasyOne Pro (New Diagnostic Design (NDD) Medical Technologies IncMultiple breath washout testing will be completed on different devices over time | Device | – |
Primary outcome
Lung Clearance Index
Time frame: Each testing session may take up to 90 minutes but most likely it will be under an hour. The MBW test will be completed at each visit and the LCI and other MBW results are obtained from data analysis post completion.
Multiple breath washout involves recording concentrations of a tracer gas whilst it is cleared from the lungs during normal tidal breathing. The tracer gas can either be an inert gas such as sulphur hexafluoride (SF6) that is washed out by room air, or resident Nitrogen (N2) that is cleared by 100% oxygen (O2). All tracer gases are traditionally cleared to 1/40th (or 2.5%) of their starting concentration. This procedure is performed in triplicate, each test will take around 5-10 minutes with wait time in-between.
3 secondary outcomes
Spirometry
Time frame: Spirometry will be completed at each visit (unless already taken at a clinical visit). Maximum 10 visits over 3 years
Disease specific Quality of life questionnaire
Time frame: The quality of life questionnaire will be completed at each visit and compared to the primary outcome at each visit. Maximum 10 visits over 3 years
Review of clinically indicated tests
Time frame: Post each visit review of clinically indicated tests will take place and compared to the primary outcome. Maximum 10 visits over 3 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2022.
- Individual participant dataSharing planned: No
Related trials for Cystic Fibrosis
Not yet recruitingObservational study
Vascular Health and Function in Cystic Fibrosis
Cystic Fibrosis, Endothelial Dysfunction
Not yet recruitingPhase 4
Iron Repletion in Cystic Fibrosis
Cystic Fibrosis, Iron Deficiencies
Recruiting
Diagnostic Value of Oropharyngeal Suction and Effect of Respiratory Physiotherapy on the Detection of Microorganisms in the Pediatric Patient
Cystic Fibrosis (CF), Bronchiectasis, Non-Cystic Fibrosis
RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
RecruitingObservational study
PERCE-MUCO: Perceptions and Expectations of People With Cystic Fibrosis Regarding a Change in Treatment
Cystic Fibrosis (CF)
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.
Jane C Davies, Professor principal investigator
Imperial College 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 Imperial College London 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 9 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: status not confirmed, as of the sponsor's last update on 6 May 2022. 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 Katie J Bayfield (02075947949, k.bayfield@imperial.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Lung Clearance Index (time frame: Each testing session may take up to 90 minutes but most likely it will be under an hour. The MBW test will be completed at each visit and the LCI and other MBW results are obtained from data analysis post completion.). 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 6 May 2022.