NCT02606487

Brief Summary

The investigators aim to assess whether pulmonary MRI (hyperpolarised 129Xe ventilation imaging \[Xe-MRI\]) can detect changes in ventilation defects in patients with CF before and after treatment for a pulmonary exacerbation. The investigators will determine whether changes seen using pulmonary Xe-MRI are associated with changes in pulmonary function (spirometry, lung volumes, lung clearance index \[LCI\]) in patients with CF before and after pulmonary exacerbation.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Nov 2015

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2015

Completed
12 days until next milestone

First Submitted

Initial submission to the registry

November 13, 2015

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 17, 2015

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2017

Completed
Last Updated

October 4, 2018

Status Verified

October 1, 2018

Enrollment Period

2 years

First QC Date

November 13, 2015

Last Update Submit

October 3, 2018

Conditions

Cystic Fibrosis

Keywords

CFExacerbationHyerpolarized xenon MRIPediatricsLung Clearance Index

Outcome Measures

Primary Outcomes (4)

  • Ventilation Defect Percentage (VDP)

    VDP within 48h of initiation of inpatient treatment

    Pre-treatment

  • Ventilation Defect Percentage (VDP)

    VDP within 48h of completion of inpatient treatment

    Post-treatment - within 48h of completion of inpatient treatment

  • Lung Clearance Index (LCI)

    LCI within 48h of initiation of inpatient treatment

    Pre-treatment

  • Lung Clearance Index (LCI)

    LCI within 48h of completion of inpatient treatment

    Post-treatment - within 48h of completion of inpatient treatment

Secondary Outcomes (2)

  • Pulmonary function tests (PFTs)

    Pre-treatment

  • Pulmonary function tests (PFTs)

    Post-treatment - within 48h of completion of inpatient treatment

Study Arms (1)

CF pulmonary exacerbation group

Patients with cystic fibrosis admitted for inpatient treatment of a pulmonary exacerbation

Eligibility Criteria

Age8 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

Patients aged 8-18 with cystic fibrosis admitted for inpatient treatment with a clinical diagnosis of pulmonary exacerbation

You may qualify if:

  • Diagnosis of CF as defined by two or more clinical features of CF and a documented sweat chloride \> 60mEq/L by quantitative pilocarpine iontophoresis test or a genotype showing two well characterized disease causing mutations
  • Informed consent and verbal assent (as appropriate) provided by the subject's parent or legal guardian and the subject
  • Ages 8-18 years and able to perform reproducible spirometry and achieve a breath hold duration sufficient for MRI acquisition
  • Admission to the Hospital for Sick Children for a pulmonary exacerbation (based on clinical or pulmonary function assessment). Children who will be admitted and then discharged on home IV antibiotics may also be included in this study.

You may not qualify if:

  • Inability to perform reproducible pulmonary function tests (spirometry, plethysmography or lung clearance index) or perform a breath-hold of sufficient duration for MRI acquisition
  • Medical instability that would preclude the ability to undergo the required investigations
  • FEV1 % predicted \< 40%
  • Use of supplementary oxygen
  • Severe claustrophobia
  • Pregnancy or lactation
  • Presence of metal implants or other contraindications to MRI

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (7)

  • O'Sullivan BP, Freedman SD. Cystic fibrosis. Lancet. 2009 May 30;373(9678):1891-904. doi: 10.1016/S0140-6736(09)60327-5. Epub 2009 May 4.

    PMID: 19403164BACKGROUND

  • Robinson PD, Latzin P, Verbanck S, Hall GL, Horsley A, Gappa M, Thamrin C, Arets HG, Aurora P, Fuchs SI, King GG, Lum S, Macleod K, Paiva M, Pillow JJ, Ranganathan S, Ratjen F, Singer F, Sonnappa S, Stocks J, Subbarao P, Thompson BR, Gustafsson PM. Consensus statement for inert gas washout measurement using multiple- and single- breath tests. Eur Respir J. 2013 Mar;41(3):507-22. doi: 10.1183/09031936.00069712. Epub 2013 Feb 8.

    PMID: 23397305BACKGROUND

  • Davies J, Sheridan H, Bell N, Cunningham S, Davis SD, Elborn JS, Milla CE, Starner TD, Weiner DJ, Lee PS, Ratjen F. Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med. 2013 Oct;1(8):630-638. doi: 10.1016/S2213-2600(13)70182-6. Epub 2013 Sep 10.

    PMID: 24461666BACKGROUND

  • Horsley AR, Davies JC, Gray RD, Macleod KA, Donovan J, Aziz ZA, Bell NJ, Rainer M, Mt-Isa S, Voase N, Dewar MH, Saunders C, Gibson JS, Parra-Leiton J, Larsen MD, Jeswiet S, Soussi S, Bakar Y, Meister MG, Tyler P, Doherty A, Hansell DM, Ashby D, Hyde SC, Gill DR, Greening AP, Porteous DJ, Innes JA, Boyd AC, Griesenbach U, Cunningham S, Alton EW. Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation. Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.

    PMID: 23396354BACKGROUND

  • Yammine S, Bigler A, Casaulta C, Singer F, Latzin P. Reasons for heterogeneous change in LCI in children with cystic fibrosis after antibiotic treatment. Thorax. 2014 Feb;69(2):183. doi: 10.1136/thoraxjnl-2013-204283. Epub 2013 Aug 29. No abstract available.

    PMID: 23988751BACKGROUND

  • Shukla Y, Wheatley A, Kirby M, Svenningsen S, Farag A, Santyr GE, Paterson NA, McCormack DG, Parraga G. Hyperpolarized 129Xe magnetic resonance imaging: tolerability in healthy volunteers and subjects with pulmonary disease. Acad Radiol. 2012 Aug;19(8):941-51. doi: 10.1016/j.acra.2012.03.018. Epub 2012 May 15.

    PMID: 22591724BACKGROUND

  • Munidasa S, Couch MJ, Rayment JH, Voskrebenzev A, Seethamraju R, Vogel-Claussen J, Ratjen F, Santyr G. Free-breathing MRI for monitoring ventilation changes following antibiotic treatment of pulmonary exacerbations in paediatric cystic fibrosis. Eur Respir J. 2021 Apr 15;57(4):2003104. doi: 10.1183/13993003.03104-2020. Print 2021 Apr. No abstract available.

    PMID: 33303537DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Officials

  • Felix Ratjen, MD PhD

    The Hospital for Sick Children

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Division Head, Respiratory Medicine

Study Record Dates

First Submitted

November 13, 2015

First Posted

November 17, 2015

Study Start

November 1, 2015

Primary Completion

November 1, 2017

Study Completion

November 1, 2017

Last Updated

October 4, 2018

Record last verified: 2018-10

Locations

CA(1)