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Utilizing Hyperpolarized 129Xe Magnetic Resonance Imaging in Children With Primary Ciliary Dyskinesia (PCD MRI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04858191
Recruitment Status : Not yet recruiting
First Posted : April 26, 2021
Last Update Posted : September 21, 2021
Sponsor:
Collaborator:
Provincial Health Services Authority
Information provided by (Responsible Party):
Felix Ratjen, The Hospital for Sick Children

Study Description
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Brief Summary:
This study investigates the use of hyperpolarized 129Xe magnetic resonance imaging (MRI) in children with primary ciliary dyskinesia (PCD) in detecting ventilation defects. The investigators will establish the feasibility and reliability of this test and how it changes compared to other pulmonary function tests.

Condition or disease
Primary Ciliary Dyskinesia

Detailed Description:

Primary Ciliary Dyskinesia (PCD) is an autosomal recessive inherited disorder caused by defects in ciliary structure and/or function. Prevention or delaying disease progression requires medical therapies and routine lung function monitoring, with the goal of early initiation of medical therapies. Of course, this is contingent on recognizing early lung disease.

Current investigations for monitoring lung disease include pulmonary function tests (PFT), chest x rays and chest CTs. But each of these modalities are either not sensitive enough or expose the patient to ionizing radiation.

The investigators believe that hyperpolarized 129Xe MRI (HP Xe-MRI), new imaging modality, will be more sensitive then current tests and also avoid the need for ionizing radiation. To evaluate this, The investigators will compare HP Xe-MRI to PFT, when the patient is well and during a pulmonary exacerbation that is being treated.

Study Design
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Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Utilizing Hyperpolarized 129Xe Magnetic Resonance Imaging in Children With Primary Ciliary Dyskinesia
Estimated Study Start Date : October 2021
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: MRI Scans

Groups and Cohorts
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Group/Cohort
Pediatric PCD
Pediatric participants with PCD


Outcome Measures
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Primary Outcome Measures :
  1. Ventilation Defect Percentage (VDP) [ Time Frame: Within 1 year of study initiation ]
    Reliability; initial test

  2. Ventilation Defect Percentage (VDP) [ Time Frame: Within 1 week of initial test ]
    Reliability; re-test

  3. Ventilation Defect Percentage (VDP) [ Time Frame: Within 48 hours of pulmonary exacerbation diagnosis ]
    VDP within 48h of pulmonary exacerbation diagnosis

  4. Ventilation Defect Percentage (VDP) [ Time Frame: Within 48 hours of antibiotic completion ]
    VDP within 48h of antibiotic completion


Secondary Outcome Measures :
  1. Pulmonary function tests (PFTs) [ Time Frame: Within 1 year of study initiation ]
    Reliability; initial test

  2. Pulmonary function tests (PFTs) [ Time Frame: Within 1 week of initial test ]
    Reliability; re-test

  3. Pulmonary function tests (PFTs) [ Time Frame: Within 48 hours of pulmonary exacerbation diagnosis ]
    PFT within 48h of pulmonary exaction diagnosis

  4. Pulmonary function tests (PFTs) [ Time Frame: Within 48 hours of antibiotic completion ]
    PFT within 48h of antibiotic completion


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients aged 6-18 with primary ciliary dyskinesia
Criteria

Inclusion Criteria

  • Diagnosis of PCD as having either (i) biallelic mutations in known PCD genes or (ii) classic transmission electron microscopy structural ciliary defect
  • Informed consent and verbal assent (as appropriate) provided by the participant's parent or legal guardian and the participant
  • Ages 6-18 years and able to perform reproducible spirometry and achieve a breath hold duration sufficient for MRI acquisition

Exclusion Criteria

  • Any other cardiac or respiratory disease
  • Inability to perform a breath-hold of adequate duration for MRI acquisition
  • Medical instability that would preclude the ability to undergo the required investigations
  • FEV1 % predicted <40% on any PFT within last 2 months at time of consent
  • Use of supplementary oxygen
  • Severe claustrophobia
  • Pregnancy or lactation
  • Presence of metal implants or other MRI contraindications
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04858191


Contacts
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Contact: Wallace Wee, MD 416-813-5587 wallace.wee@sickkids.ca
Contact: Michael Sawras 416-813-5587 michael.sawras@sickkids.ca

Locations
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Canada, Ontario
Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8
Contact: Wallace Wee, MD    416-813-5587    wallace.wee@sickkids.ca   
Contact: Michael Sawras    416-813-5587      
Principal Investigator: Felix Ratjen, MD         
Sponsors and Collaborators
The Hospital for Sick Children
Provincial Health Services Authority
Investigators
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Principal Investigator: Felix Ratjen, MD, PhD, FRCP(C), FERS The Hospital for Sick Children
More Information
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Responsible Party: Felix Ratjen, Principle investigator, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT04858191    
Other Study ID Numbers: 1000068639
First Posted: April 26, 2021    Key Record Dates
Last Update Posted: September 21, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Data will be shared between two participating sites for this study. A data transfer agreement will be created and implemented to ensure smooth transfer of data
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: The data will be available after enrolling the first participant and will be available for the duration of the study.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Felix Ratjen, The Hospital for Sick Children:
Xenon MRI
Lung
Pulmonary Exacerbation
Additional relevant MeSH terms:
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Ciliary Motility Disorders
Dyskinesias
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
 Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Ciliopathies
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn