Primary Ciliary DyskinesiaPCD MRINCT04858191
Utilizing Hyperpolarized 129Xe Magnetic Resonance Imaging in Children With Primary Ciliary Dyskinesia
An observational study of Primary Ciliary Dyskinesia, run by The Hospital for Sick Children at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Primary Ciliary Dyskinesia
- How many people
- 16 participants
- Where
- 1 siteCanada
- Study dates
- 1 Sep 2021to 30 Jun 2023
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients aged 6-18 with primary ciliary dyskinesia
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- Xenon MRI, Lung, Pulmonary Exacerbation
- Medical subject headings
- Ciliary Motility Disorders
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 |
|---|---|---|
| Pediatric PCD | Pediatric participants with PCD |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ventilation Defect Percentage (VDP)
Reliability; initial test
Measured: Within 1 year of study initiation
Ventilation Defect Percentage (VDP)
Reliability; re-test
Measured: Within 1 week of initial test
Ventilation Defect Percentage (VDP)
VDP within 48h of pulmonary exacerbation diagnosis
Measured: Within 48 hours of pulmonary exacerbation diagnosis
Ventilation Defect Percentage (VDP)
VDP within 48h of antibiotic completion
Measured: Within 48 hours of antibiotic completion
Secondary outcomes (4)
Pulmonary function tests (PFTs)
Reliability; initial test
Measured: Within 1 year of study initiation
Pulmonary function tests (PFTs)
Reliability; re-test
Measured: Within 1 week of initial test
Pulmonary function tests (PFTs)
PFT within 48h of pulmonary exaction diagnosis
Measured: Within 48 hours of pulmonary exacerbation diagnosis
Pulmonary function tests (PFTs)
PFT within 48h of antibiotic completion
Measured: Within 48 hours of antibiotic completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2021
- Study start1 Sep 2021
- Primary completion30 Jun 2023
- Study completion30 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Ciliary DyskinesiaStudies 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
- Other
- Time perspective
- Prospective
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Collaborators
- Provincial Health Services Authority British Columbia
- Responsible party
- Principal investigator, Felix Ratjen, The Hospital for Sick Children
- Investigators
- Felix Ratjen, MD, PhD, FRCP(C), FERS, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04858191
- Sponsor's protocol ID
- 1000068639
- Acronym
- PCD MRI
- Status last verified
- Oct 2023
- First submitted
- 25 Mar 2021
- Last updated
- 23 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
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
- Utilizing Hyperpolarized 129Xe Magnetic Resonance Imaging in Children With Primary Ciliary Dyskinesia
- ClinicalTrials.gov ID
- NCT04858191
- Acronym / sponsor ID
- PCD MRI / 1000068639
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- Xenon MRI, Lung, Pulmonary Exacerbation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Collaborators
- Provincial Health Services Authority British Columbia
- Study start
- 1 Sep 2021 (actual)
- Primary completion
- 30 Jun 2023 (actual)
- Study completion
- 30 Jun 2023 (actual)
- First posted
- 26 Apr 2021 (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.
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.
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
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric PCD | – | –Pediatric participants with PCD |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients aged 6-18 with primary ciliary dyskinesia
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (8 listed)
- 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
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
Ventilation Defect Percentage (VDP)
Time frame: Within 1 year of study initiation
Reliability; initial test
Ventilation Defect Percentage (VDP)
Time frame: Within 1 week of initial test
Reliability; re-test
Ventilation Defect Percentage (VDP)
Time frame: Within 48 hours of pulmonary exacerbation diagnosis
VDP within 48h of pulmonary exacerbation diagnosis
Ventilation Defect Percentage (VDP)
Time frame: Within 48 hours of antibiotic completion
VDP within 48h of antibiotic completion
4 secondary outcomes
Pulmonary function tests (PFTs)
Time frame: Within 1 year of study initiation
Pulmonary function tests (PFTs)
Time frame: Within 1 week of initial test
Pulmonary function tests (PFTs)
Time frame: Within 48 hours of pulmonary exacerbation diagnosis
Pulmonary function tests (PFTs)
Time frame: Within 48 hours of antibiotic completion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2023.
- Individual participant dataSharing planned: Yes. 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
Related trials for Primary Ciliary Dyskinesia
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Comparison of Individuals With Primary Ciliary Dyskinesia and Healthy Controls
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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, FRCP(C), FERS 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 Primary Ciliary Dyskinesia, 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 3 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: finished, 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ventilation Defect Percentage (VDP) (time frame: Within 1 year of study initiation). The study lists 4 primary outcomes in all. 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 23 Oct 2023; AzTrials last refreshed its copy on 2 Nov 2026.