Measures of Respiratory Health (MRH)

NCT ID: NCT02657837

Last Updated: 2023-10-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-01-31

Study Completion Date

2024-12-01

Brief Summary

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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

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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

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Cystic Fibrosis Asthma Sickle Cell Anemia Bronchiolitis Obliterans

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Cystic Fibrosis

Children 2.5 to 18 years old with confirmed diagnosis of cystic fibrosis

No interventions assigned to this group

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.

No interventions assigned to this group

Healthy Children

Children and adults 2.5 to 30 years old with no history of chronic disease

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

1. 2.5 - 18 years of age at enrolment
2. 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:

1. A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
2. A documented genotype with two disease-causing mutations in the CFTR gene
3. Informed consent by participant, parent, or legal guardian
4. Ability to perform technically acceptable MBW measurements


1. MD diagnosed lung disease and/or attending the Pulmonary Function Laboratory
2. 2.5 - 18 years of age at enrollment
3. Informed consent by participant, parent, or legal guardian
4. Ability to perform technically acceptable MBW measurements


1. 2.5 - 30 years of age at enrollment
2. Informed consent by participant, parent, or legal guardian
3. Ability to perform technically acceptable MBW measurements

Exclusion Criteria

1. Physical findings at screening that would compromise the safety of the participant as judged by the patient's most responsible physician
2. Requirement of supplementary oxygen to maintain an oxygen saturation above 95%

Participants with other respiratory disease


1. Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
2. Requirement of supplementary oxygen to maintain an oxygen saturation above 95%

Healthy Participants


1. Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
2. Evidence of a chronic disease process such as lung disease
Minimum Eligible Age

30 Months

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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The Hospital for Sick Children

OTHER

Sponsor Role lead

Responsible Party

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Felix Ratjen

Division Head, Respiratory Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Felix Ratjen, MD PhD

Role: PRINCIPAL_INVESTIGATOR

The Hospital for Sick Children

Locations

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The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Countries

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Canada

Central Contacts

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Felix Ratjen, MD PhD

Role: CONTACT

416-813-7654 ext. 228472

Sanja Stanojevic, PhD

Role: CONTACT

416-813-7654 ext. 328077

Facility Contacts

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Felix Ratjen, MD PhD

Role: primary

416-813-7654 ext. 228472

References

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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.

Reference Type BACKGROUND
PMID: 24429095 (View on PubMed)

Ranganathan 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.

Reference Type BACKGROUND
PMID: 11747924 (View on PubMed)

Sly 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.

Reference Type BACKGROUND
PMID: 19372250 (View on PubMed)

Horsley 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.

Reference Type BACKGROUND
PMID: 19246184 (View on PubMed)

Gustafsson 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.

Reference Type BACKGROUND
PMID: 14680088 (View on PubMed)

Aurora 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.

Reference Type BACKGROUND
PMID: 15563707 (View on PubMed)

Gustafsson 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.

Reference Type BACKGROUND
PMID: 17675316 (View on PubMed)

Owens 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.

Reference Type BACKGROUND
PMID: 21422040 (View on PubMed)

Hall 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.

Reference Type BACKGROUND
PMID: 21886842 (View on PubMed)

Aurora 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.

Reference Type BACKGROUND
PMID: 20935113 (View on PubMed)

Other Identifiers

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1000051399

Identifier Type: -

Identifier Source: org_study_id

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