Utilizing Hyperpolarized 129Xe Magnetic Resonance Imaging... | NCT04858191

Studies
Utilizing Hyperpolarized 129Xe Magnetic Resonance Imaging in Children With Primary Ciliary Dyskinesia

NCT04858191

Sponsor

The Hospital for Sick Children

Status

Completed

Last Update Posted

Oct 23, 2023Actual

Enrollment

16Actual

Phase

Not provided

Conditions

Primary Ciliary Dyskinesia

Interventions

Not provided

Countries

Canada

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Oct 2023

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 1, 2021Actual

Primary Completion Date

Jun 30, 2023Actual

Completion Date

Jun 30, 2023Actual

First Submitted Date

Mar 25, 2021

First Submission Date that Met QC Criteria

Apr 20, 2021

First Posted Date

Apr 26, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Oct 19, 2023

Last Update Posted Date

Oct 23, 2023Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Felix Ratjen, Principle investigator, The Hospital for Sick ChildrenPrincipal Investigator

Lead Sponsor

The Hospital for Sick ChildrenOTHER

Collaborators

Name	Class
Provincial Health Services Authority British Columbia	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

16Actual

Label	Type	Description	Intervention Names
Pediatric PCD		Pediatric participants with PCD

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Ventilation Defect Percentage (VDP)	Reliability; initial test	Within 1 year of study initiation
Ventilation Defect Percentage (VDP)	Reliability; re-test	Within 1 week of initial test
Ventilation Defect Percentage (VDP)	VDP within 48h of pulmonary exacerbation diagnosis	Within 48 hours of pulmonary exacerbation diagnosis
Ventilation Defect Percentage (VDP)	VDP within 48h of antibiotic completion	Within 48 hours of antibiotic completion

Secondary Outcomes

Measure	Description	Time Frame
Pulmonary function tests (PFTs)	Reliability; initial test	Within 1 year of study initiation
Pulmonary function tests (PFTs)	Reliability; re-test

Other Outcomes

Not provided

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Patients aged 6-18 with primary ciliary dyskinesia

Sampling Method

Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Felix Ratjen, MD, PhD, FRCP(C), FERS	The Hospital for Sick Children	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital for Sick Children		Toronto	Ontario	M5G1X8	Canada

IPD Sharing Statement Module

Plan to Share IPD

Yes

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

Types

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.

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D002925	Ciliary Motility Disorders

Ancestor Terms

ID	Term
D012140	Respiratory Tract Diseases
D010038	Otorhinolaryngologic Diseases
D000072661	Ciliopathies
D000015	Abnormalities, Multiple

Browse Leaves

Not provided

Browse Branches

Not provided