Cystic FibrosisMRHNCT02657837
Measures of Respiratory Health (MRH)
An observational study of Cystic Fibrosis and Asthma, run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
The Lung Clearance Index, measured by multiple breath washout, is a measure of lung function that is considered a research tool in Canada as the device used to measure it is not approved by Health Canada. Read more about this study
- Who is it for?
- Ages 30 months to 30 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Asthma, Sickle Cell Anemia
- How many people
- 300 participants
- Where
- 1 siteCanada
- Study dates
- Jan 2016to 1 Dec 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 30 months to 30 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Participants who meet all of the inclusion and none of the exclusion criteria will be eligible for participation in this study. Participants will include individuals with CF, individuals with other respiratory disease and healthy individuals.
Who may be able to join
- 2.5 - 18 years of age at enrolment
- Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:
- A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
- A documented genotype with two disease-causing mutations in the CFTR gene
- Informed consent by participant, parent, or legal guardian
- Ability to perform technically acceptable MBW measurements
Show all 13Show fewer
- MD diagnosed lung disease and/or attending the Pulmonary Function Laboratory
- 2.5 - 18 years of age at enrollment
- Informed consent by participant, parent, or legal guardian
- Ability to perform technically acceptable MBW measurements
- 2.5 - 30 years of age at enrollment
- Informed consent by participant, parent, or legal guardian
- Ability to perform technically acceptable MBW measurements
Who cannot join
- Physical findings at screening that would compromise the safety of the participant as judged by the patient's most responsible physician
- Requirement of supplementary oxygen to maintain an oxygen saturation above 95%
- Participants with other respiratory disease
- Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
- Requirement of supplementary oxygen to maintain an oxygen saturation above 95%
- Healthy Participants
- Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
- Evidence of a chronic disease process such as lung disease
Trial locations and contacts
1 site in 1 country.
Canada1 site, 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Felix Ratjen, MD PhD, 416-813-7654 ext. 228472, felix.ratjen@sickkids.ca
Hartmut Grasemann, MD PhD
About this study
Official title: Measures of Respiratory Health
The Lung Clearance Index, measured by multiple breath washout, is a measure of lung function that is considered a research tool in Canada as the device used to measure it is not approved by Health Canada. The study will assess lung function in patients undergoing routine lung function testing for clinical indications (Cystic Fibrosis and Other Respiratory Diseases). In addition, healthy controls of different ages will be asked to perform this lung function test to gain reference data that can be used to interpret LCI in patients with lung disease.
Read the detailed description
Functional abnormalities associated with lung disease such as cystic fibrosis (CF) occur in early childhood, but have historically gone undetected until the onset of clinical symptoms, at which point irreversible lung damage may have already occurred (1-3). Consequently, over the last ten years the focus of clinical care has shifted to early intervention and prevention of these structural changes. To facilitate early intervention there is a pressing need for surrogate markers of early obstructive lung disease that are also sensitive enough to detect treatment effects (4).
The Lung Clearance Index (LCI) is a promising marker for detecting early lung disease. The LCI is measured by multiple breath washout (MBW) and is an indicator of ventilation inhomogeneity. MBW is performed during quiet tidal breathing and requires minimal effort from patients. It is feasible in all age groups when adaptions are made for younger children.
While there are Health Canada licensed washout systems available that can determine LCI; these devices have not been adequately validated; thus their use in routine lung function testing is controversial. As part of an international effort to validate multiple breath washout testing to measure LCI, the specific device is licensed in Europe, but as of yet is not Health Canada approved. Thus, testing with this device is considered research. This study will utilize technology to assess lung function in patients undergoing routine lung function testing for clinical indications. In addition, healthy controls of different ages will be asked to perform this lung function test to gain reference data that can be used to interpret LCI in patients with lung disease.
- Conditions
- Cystic Fibrosis, Asthma, Sickle Cell Anemia, Bronchiolitis Obliterans
- Keywords
- CF, Multiple Breath Washout, Lung Clearance Index, Pediatrics
- Medical subject headings
- Cystic Fibrosis, Asthma, Anemia, Sickle Cell, Bronchiolitis Obliterans
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 |
|---|---|---|
| Cystic Fibrosis | Children 2.5 to 18 years old with confirmed diagnosis of cystic fibrosis | |
| Children with other respiratory disease | Children 2.5 to 18 years old with confirmed diagnosis of respiratory disease including but not limited to asthma, transplant, and sickle cell anemia. | |
| Healthy Children | Children and adults 2.5 to 30 years old with no history of chronic disease |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients with an abnormal Lung Clearance Index (>7.5).
Single time point measurements obtained in enrolled subjects
Measured: Day 1
Secondary outcomes (1)
. Proportion of patients with an abnormal pulmonary function tests based on spirometry.
Single time point measurements obtained in enrolled subjects
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2016 (estimated)
- Study startJan 2016
- Primary completion1 Dec 2024 (estimated)
- Study completion1 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by The Hospital for Sick ChildrenThe 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
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Felix Ratjen, The Hospital for Sick Children
- Investigators
- Felix Ratjen, MD PhD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02657837
- Sponsor's protocol ID
- 1000051399
- Acronym
- MRH
- Status last verified
- Oct 2023
- First submitted
- 8 Jan 2016
- Last updated
- 23 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
Publications
Grasemann H, Ratjen F. Early lung disease in cystic fibrosis. Lancet Respir Med. 2013 Apr;1(2):148-57. doi: 10.1016/S2213-2600(13)70026-2. Epub 2013 Mar 12.
PubMed 24429095Ranganathan SC, Dezateux C, Bush A, Carr SB, Castle RA, Madge S, Price J, Stroobant J, Wade A, Wallis C, Stocks J; London Collaborative Cystic Fibrosis Group. Airway function in infants newly diagnosed with cystic fibrosis. Lancet. 2001 Dec 8;358(9297):1964-5. doi: 10.1016/s0140-6736(01)06970-7.
PubMed 11747924Sly PD, Brennan S, Gangell C, de Klerk N, Murray C, Mott L, Stick SM, Robinson PJ, Robertson CF, Ranganathan SC; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST-CF). Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. Am J Respir Crit Care Med. 2009 Jul 15;180(2):146-52. doi: 10.1164/rccm.200901-0069OC. Epub 2009 Apr 16.
PubMed 19372250Horsley A. Lung clearance index in the assessment of airways disease. Respir Med. 2009 Jun;103(6):793-9. doi: 10.1016/j.rmed.2009.01.025. Epub 2009 Feb 25.
PubMed 19246184Gustafsson PM, Aurora P, Lindblad A. Evaluation of ventilation maldistribution as an early indicator of lung disease in children with cystic fibrosis. Eur Respir J. 2003 Dec;22(6):972-9. doi: 10.1183/09031936.03.00049502.
PubMed 14680088Aurora P, Gustafsson P, Bush A, Lindblad A, Oliver C, Wallis CE, Stocks J. Multiple breath inert gas washout as a measure of ventilation distribution in children with cystic fibrosis. Thorax. 2004 Dec;59(12):1068-73. doi: 10.1136/thx.2004.022590.
PubMed 15563707Gustafsson PM, De Jong PA, Tiddens HA, Lindblad A. Multiple-breath inert gas washout and spirometry versus structural lung disease in cystic fibrosis. Thorax. 2008 Feb;63(2):129-34. doi: 10.1136/thx.2007.077784. Epub 2007 Aug 3.
PubMed 17675316Owens CM, Aurora P, Stanojevic S, Bush A, Wade A, Oliver C, Calder A, Price J, Carr SB, Shankar A, Stocks J; London Cystic Fibrosis Collaboration. Lung Clearance Index and HRCT are complementary markers of lung abnormalities in young children with CF. Thorax. 2011 Jun;66(6):481-8. doi: 10.1136/thx.2010.150375. Epub 2011 Mar 21.
PubMed 21422040Hall GL, Logie KM, Parsons F, Schulzke SM, Nolan G, Murray C, Ranganathan S, Robinson P, Sly PD, Stick SM; AREST CF; Berry L, Garratt L, Massie J, Mott L, Poreddy S, Simpson S. Air trapping on chest CT is associated with worse ventilation distribution in infants with cystic fibrosis diagnosed following newborn screening. PLoS One. 2011;6(8):e23932. doi: 10.1371/journal.pone.0023932. Epub 2011 Aug 19.
PubMed 21886842Aurora P, Stanojevic S, Wade A, Oliver C, Kozlowska W, Lum S, Bush A, Price J, Carr SB, Shankar A, Stocks J; London Cystic Fibrosis Collaboration. Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosis. Am J Respir Crit Care Med. 2011 Mar 15;183(6):752-8. doi: 10.1164/rccm.200911-1646OC. Epub 2010 Oct 8.
PubMed 20935113
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Measures of Respiratory Health
- ClinicalTrials.gov ID
- NCT02657837
- Acronym / sponsor ID
- MRH / 1000051399
- Conditions
- Cystic Fibrosis, Asthma, Sickle Cell Anemia, Bronchiolitis Obliterans
- Keywords
- CF, Multiple Breath Washout, Lung Clearance Index, Pediatrics
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- Jan 2016
- Primary completion
- 1 Dec 2024 (estimated)
- Study completion
- 1 Dec 2024 (estimated)
- First posted
- 18 Jan 2016 (estimated)
- 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.
The Lung Clearance Index, measured by multiple breath washout, is a measure of lung function that is considered a research tool in Canada as the device used to measure it is not approved by Health Canada. The study will assess lung function in patients undergoing routine lung function testing for clinical indications (Cystic Fibrosis and Other Respiratory Diseases). In addition, healthy controls of different ages will be asked to perform this lung function test to gain reference data that can be used to interpret LCI in patients with lung disease.
Detailed description
Functional abnormalities associated with lung disease such as cystic fibrosis (CF) occur in early childhood, but have historically gone undetected until the onset of clinical symptoms, at which point irreversible lung damage may have already occurred (1-3). Consequently, over the last ten years the focus of clinical care has shifted to early intervention and prevention of these structural changes. To facilitate early intervention there is a pressing need for surrogate markers of early obstructive lung disease that are also sensitive enough to detect treatment effects (4).
The Lung Clearance Index (LCI) is a promising marker for detecting early lung disease. The LCI is measured by multiple breath washout (MBW) and is an indicator of ventilation inhomogeneity. MBW is performed during quiet tidal breathing and requires minimal effort from patients. It is feasible in all age groups when adaptions are made for younger children.
While there are Health Canada licensed washout systems available that can determine LCI; these devices have not been adequately validated; thus their use in routine lung function testing is controversial. As part of an international effort to validate multiple breath washout testing to measure LCI, the specific device is licensed in Europe, but as of yet is not Health Canada approved. Thus, testing with this device is considered research. This study will utilize technology to assess lung function in patients undergoing routine lung function testing for clinical indications. In addition, healthy controls of different ages will be asked to perform this lung function test to gain reference data that can be used to interpret LCI in patients with lung disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cystic Fibrosis | – | –Children 2.5 to 18 years old with confirmed diagnosis of cystic fibrosis |
| Children with other respiratory disease | – | –Children 2.5 to 18 years old with confirmed diagnosis of respiratory disease including but not limited to asthma, transplant, and sickle cell anemia. |
| Healthy Children | – | –Children and adults 2.5 to 30 years old with no history of chronic disease |
Who may qualify
- Ages
- 30 months to 30 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Participants who meet all of the inclusion and none of the exclusion criteria will be eligible for participation in this study. Participants will include individuals with CF, individuals with other respiratory disease and healthy individuals.
Key inclusion criteria (13 listed)
- 2.5 - 18 years of age at enrolment
- Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:
- Informed consent by participant, parent, or legal guardian
- Ability to perform technically acceptable MBW measurements
- MD diagnosed lung disease and/or attending the Pulmonary Function Laboratory
Key exclusion criteria (8 listed)
- Physical findings at screening that would compromise the safety of the participant as judged by the patient's most responsible physician
- Requirement of supplementary oxygen to maintain an oxygen saturation above 95%
- Participants with other respiratory disease
- Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
- Requirement of supplementary oxygen to maintain an oxygen saturation above 95%
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
Proportion of patients with an abnormal Lung Clearance Index (>7.5).
Time frame: Day 1
Single time point measurements obtained in enrolled subjects
1 secondary outcome
. Proportion of patients with an abnormal pulmonary function tests based on spirometry.
Time frame: Day 1
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2023.
- Background: PubMed 24429095Grasemann H, Ratjen F. Early lung disease in cystic fibrosis. Lancet Respir Med. 2013 Apr;1(2):148-57. doi: 10.1016/S2213-2600(13)70026-2. Epub 2013 Mar 12.
- Background: PubMed 11747924Ranganathan SC, Dezateux C, Bush A, Carr SB, Castle RA, Madge S, Price J, Stroobant J, Wade A, Wallis C, Stocks J; London Collaborative Cystic Fibrosis Group. Airway function in infants newly diagnosed with cystic fibrosis. Lancet. 2001 Dec 8;358(9297):1964-5. doi: 10.1016/s0140-6736(01)06970-7.
- Background: PubMed 19372250Sly PD, Brennan S, Gangell C, de Klerk N, Murray C, Mott L, Stick SM, Robinson PJ, Robertson CF, Ranganathan SC; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST-CF). Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. Am J Respir Crit Care Med. 2009 Jul 15;180(2):146-52. doi: 10.1164/rccm.200901-0069OC. Epub 2009 Apr 16.
- Background: PubMed 19246184Horsley A. Lung clearance index in the assessment of airways disease. Respir Med. 2009 Jun;103(6):793-9. doi: 10.1016/j.rmed.2009.01.025. Epub 2009 Feb 25.
- Background: PubMed 14680088Gustafsson PM, Aurora P, Lindblad A. Evaluation of ventilation maldistribution as an early indicator of lung disease in children with cystic fibrosis. Eur Respir J. 2003 Dec;22(6):972-9. doi: 10.1183/09031936.03.00049502.
- Background: PubMed 15563707Aurora P, Gustafsson P, Bush A, Lindblad A, Oliver C, Wallis CE, Stocks J. Multiple breath inert gas washout as a measure of ventilation distribution in children with cystic fibrosis. Thorax. 2004 Dec;59(12):1068-73. doi: 10.1136/thx.2004.022590.
- Background: PubMed 17675316Gustafsson PM, De Jong PA, Tiddens HA, Lindblad A. Multiple-breath inert gas washout and spirometry versus structural lung disease in cystic fibrosis. Thorax. 2008 Feb;63(2):129-34. doi: 10.1136/thx.2007.077784. Epub 2007 Aug 3.
- Background: PubMed 21422040Owens CM, Aurora P, Stanojevic S, Bush A, Wade A, Oliver C, Calder A, Price J, Carr SB, Shankar A, Stocks J; London Cystic Fibrosis Collaboration. Lung Clearance Index and HRCT are complementary markers of lung abnormalities in young children with CF. Thorax. 2011 Jun;66(6):481-8. doi: 10.1136/thx.2010.150375. Epub 2011 Mar 21.
- Background: PubMed 21886842Hall GL, Logie KM, Parsons F, Schulzke SM, Nolan G, Murray C, Ranganathan S, Robinson P, Sly PD, Stick SM; AREST CF; Berry L, Garratt L, Massie J, Mott L, Poreddy S, Simpson S. Air trapping on chest CT is associated with worse ventilation distribution in infants with cystic fibrosis diagnosed following newborn screening. PLoS One. 2011;6(8):e23932. doi: 10.1371/journal.pone.0023932. Epub 2011 Aug 19.
- Background: PubMed 20935113Aurora P, Stanojevic S, Wade A, Oliver C, Kozlowska W, Lum S, Bush A, Price J, Carr SB, Shankar A, Stocks J; London Cystic Fibrosis Collaboration. Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosis. Am J Respir Crit Care Med. 2011 Mar 15;183(6):752-8. doi: 10.1164/rccm.200911-1646OC. Epub 2010 Oct 8.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
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.
Felix Ratjen, MD PhD principal investigator
The Hospital for Sick Children
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 Asthma, run by The Hospital for Sick Children at 1 site in Canada.
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 13 inclusion and 8 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 23 Oct 2023. 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 Canada. 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 Felix Ratjen, MD PhD (416-813-7654 ext. 228472, felix.ratjen@sickkids.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of patients with an abnormal Lung Clearance Index (>7.5). (time frame: Day 1). 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 NCT02657837, which also lists 10 linked publications.
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 23 Oct 2023.