Cystic FibrosisNCT04793867
Regional Phenotyping of CF and Non-CF Bronchiectasis
An observational study of Cystic Fibrosis and Non-CF Bronchiectasis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The Investigators propose to study pediatric subjects who are diagnosed with cystic fibrosis (CF) and patients with non-CF bronchiectasis, with the goal of developing markers of CF lung disease severity, progression, and therapy response. Read more about this study
- Who is it for?
- Ages 5 to 100 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Non-CF Bronchiectasis
- What is being tested?
- Xenon
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Feb 2021to 31 Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 5 to 100 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: CF and Non-CF Bronchiectasis patients
Who may be able to join
- CF Patients: Diagnosis of CF based on sweat chloride >60 mMol/l
- Presence of two disease causing CFTR mutations, or end organ manifestations of disease.
- Age minimum 5 years.
- Care provided by the CCHMC CF Care Center or other regional CF Care Centers if required to achieve recruitment goals.
- Subjects 5 years of age and older with no known history of cardiopulmonary disease.
Who cannot join
- Patients meeting standard MRI exclusions criteria (non-MRI-compatible metal implants, claustrophobia, etc.)
- Pregnancy or lactation.
- Patients meeting standard MRI exclusions criteria (non-MRI-compatible metal implants, claustrophobia, etc.)
- Pregnancy or lactation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Penny New
Cincinnati, Ohio, 45229
Penny A New, BBA, 513-332-7931, penny.new@cchmc.org
Zackary Cleveland, PhD
About this study
Official title: Regional Phenotyping of Cystic Fibrosis Lung Disease and Non-CF Bronchiectasis
The Investigators propose to study pediatric subjects who are diagnosed with cystic fibrosis (CF) and patients with non-CF bronchiectasis, with the goal of developing markers of CF lung disease severity, progression, and therapy response. The Investigator's central hypothesis is that image-based markers can forecast pathophysiology prior to spirometric changes.
Read the detailed description
Specific Aim 1: Validate functional imaging markers in CF patients with normal spirometry but abnormal ventilation. The Investigators hypothesize imaging phenotype can predict lung function decline, even in CF patients with normal spirometry. The Investigators will test this hypothesis by performing annual HP 129Xe ventilation MRI in a cohort of CF patients of which a subset has normal FEV1 (≥85% predicted) at baseline.
Specific Aim 2: Determine the sensitivity and specificity of early structural remodeling in CF lung disease. The Investigator's preliminary studies show that imaging markers of structural lung remodeling can be measured via radiation-free MRI (as opposed to x-ray CT). We assess the sensitivity to identify subjects with irreversible remodeling by performing serial ultra-short UTE MRI.
In the first arm (Aims 1 \& 2) Up to 50 subjects will be recruited-approximately 25 with normal FEV1 (>85% predicted) and 25 with mild to moderate disease. All subjects will be asked to undergo longitudinal (i.e., approximately annually) 129Xe and UTE MRI, spirometry, and lung clearance index (LCI) measurement. If possible, studies will be coordinated with clinical visits to maximize recruitment and retention. Up to 50 age and sex matched control subjects (i.e., subjects with no known cardiopulmonary disorders) may also be recruited to provide a reference data set from healthy subjects for comparison.
Healthy adults, including CCHMC employees) and children >5 years old may be recruited as needed for MRI sequence development and validation.
In the second arm (Aim 3), 50 CF patients with bronchiectasis and 50 with non-CF bronchiectasis will be recruited from patients already undergoing clinical bronchoscopies. Before bronchoscopy, subjects will undergo UTE. BAL will be obtained from radiologically normal areas and regions with abnormalities. (Note, image-guided sampling from multiple sites is routine practice at CCHMC in patients with CF and non-CF bronchiectasis. As such, the proposed studies will not alter the sampling pattern in a clinically significant way or increase procedure time, and will thus add no patient risk.) Clinical BAL will be collected from these regions, and small aliquots (~500 µL) will be stored separately for proteomics. The remaining fluid will be pooled and submitted for routine clinical testing, with ~500 µL reserved for pooled proteomics. The Investigators will recruit ~10 subjects/yr from both CF and non-CF groups, who will be followed annually with UTE and proteomic analysis that will be collected under Dr. Ziady's companion protocol, titled Proteomic biomarkers of CF disease and non-CF bronchiectasis to measure prognostic markers of disease-in particular of persistent bronchiectasis. For all subjects, clinical data (e.g., age, gender, CF genotype, microbiology, therapies-including CFTR modulators-and exacerbation frequency) will be captured in a REDcap database.
- Conditions
- Cystic Fibrosis, Non-CF Bronchiectasis
- Being tested
- Xenon
- Keywords
- Cystic Fibrosis, hyperpolarized xenon, LCI, Non-CF Bronchiectasis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XenonDrug
Also called 129Xe
Subjects will be scanned on a commercial 3T whole-body MRI scanner equipped with high-performance gradient systems. Natural isotopic abundance or isotopically-enriched xenon gas (~86% 129Xe, Linde Inc.) will be used for all studies. Subjects will be positioned supine in the scanner with a 129Xe RF coil around their chests. Using conventional proton MRI and a breath-hold acquisition, "scout" images will be obtained to localize the subject for subsequent 129Xe acquisitions. 129Xe images will be acquired using the same breath-hold maneuver and a pulse sequence, covering the entire lung (acquisition time <10 s, approximately 3-mm in-plane resolution, 15-mm slice thickness or less). Additional scans (sequences) may be performed. A maximum of 4 doses of 129Xe will be given throughout the study following the calibration dose.
| Study group | Role | What they receive |
|---|---|---|
| CF and Non-CF Bronchiectasis | In the first arm (Aims 1 \& 2) Up to 50 subjects will be recruited-approximately 25 with normal FEV1 (>85% predicted) and 25 with mild to moderate disease. All subjects will be asked to undergo longitudinal (i.e., approximately annually) 129Xe and UTE MRI, spirometry, and lung clearance index (LCI) measurement. Receives: Drug: Xenon | |
| Healthy Subjects | Up to 50 age and sex matched control subjects (i.e., subjects with no known cardiopulmonary disorders) may also be recruited to provide a reference data set from healthy subjects for comparison. Receives: Drug: Xenon |
What is being measured
The primary outcome is the main question the study is designed to answer.
Correlation of ventilated volume predictions obtained with 129Xe MRI vs 1H MRI
Quantify the measurement agreement between the ventilated volume of CF and Non-CF Bronchiectasis lungs vs healthy lungs
Measured: Annually for 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2021
- Study start8 Feb 2021
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zackary I. Cleveland, PhD, Children's Hospital Medical Center, Cincinnati
- Investigators
- Zackary I Cleveland, PhD, Principal investigator, CCHMC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04793867
- Sponsor's protocol ID
- 2020-0814
- Status last verified
- Oct 2026
- First submitted
- 23 Feb 2021
- Last updated
- 7 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Regional Phenotyping of Cystic Fibrosis Lung Disease and Non-CF Bronchiectasis
- ClinicalTrials.gov ID
- NCT04793867
- Acronym / sponsor ID
- 2020-0814
- Conditions
- Cystic Fibrosis, Non-CF Bronchiectasis
- Keywords
- Cystic Fibrosis, hyperpolarized xenon, LCI, Non-CF Bronchiectasis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 8 Feb 2021 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 11 Mar 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.
The Investigators propose to study pediatric subjects who are diagnosed with cystic fibrosis (CF) and patients with non-CF bronchiectasis, with the goal of developing markers of CF lung disease severity, progression, and therapy response. The Investigator's central hypothesis is that image-based markers can forecast pathophysiology prior to spirometric changes.
Detailed description
Specific Aim 1: Validate functional imaging markers in CF patients with normal spirometry but abnormal ventilation. The Investigators hypothesize imaging phenotype can predict lung function decline, even in CF patients with normal spirometry. The Investigators will test this hypothesis by performing annual HP 129Xe ventilation MRI in a cohort of CF patients of which a subset has normal FEV1 (≥85% predicted) at baseline.
Specific Aim 2: Determine the sensitivity and specificity of early structural remodeling in CF lung disease. The Investigator's preliminary studies show that imaging markers of structural lung remodeling can be measured via radiation-free MRI (as opposed to x-ray CT). We assess the sensitivity to identify subjects with irreversible remodeling by performing serial ultra-short UTE MRI.
In the first arm (Aims 1 \& 2) Up to 50 subjects will be recruited-approximately 25 with normal FEV1 (>85% predicted) and 25 with mild to moderate disease. All subjects will be asked to undergo longitudinal (i.e., approximately annually) 129Xe and UTE MRI, spirometry, and lung clearance index (LCI) measurement. If possible, studies will be coordinated with clinical visits to maximize recruitment and retention. Up to 50 age and sex matched control subjects (i.e., subjects with no known cardiopulmonary disorders) may also be recruited to provide a reference data set from healthy subjects for comparison.
Healthy adults, including CCHMC employees) and children >5 years old may be recruited as needed for MRI sequence development and validation.
In the second arm (Aim 3), 50 CF patients with bronchiectasis and 50 with non-CF bronchiectasis will be recruited from patients already undergoing clinical bronchoscopies. Before bronchoscopy, subjects will undergo UTE. BAL will be obtained from radiologically normal areas and regions with abnormalities. (Note, image-guided sampling from multiple sites is routine practice at CCHMC in patients with CF and non-CF bronchiectasis. As such, the proposed studies will not alter the sampling pattern in a clinically significant way or increase procedure time, and will thus add no patient risk.) Clinical BAL will be collected from these regions, and small aliquots (~500 µL) will be stored separately for proteomics. The remaining fluid will be pooled and submitted for routine clinical testing, with ~500 µL reserved for pooled proteomics. The Investigators will recruit ~10 subjects/yr from both CF and non-CF groups, who will be followed annually with UTE and proteomic analysis that will be collected under Dr. Ziady's companion protocol, titled Proteomic biomarkers of CF disease and non-CF bronchiectasis to measure prognostic markers of disease-in particular of persistent bronchiectasis. For all subjects, clinical data (e.g., age, gender, CF genotype, microbiology, therapies-including CFTR modulators-and exacerbation frequency) will be captured in a REDcap database.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CF and Non-CF Bronchiectasis | – | Drug: XenonIn the first arm (Aims 1 \& 2) Up to 50 subjects will be recruited-approximately 25 with normal FEV1 (>85% predicted) and 25 with mild to moderate disease. All subjects will be asked to undergo longitudinal (i.e., approximately annually) 129Xe and UTE MRI, spirometry, and lung clearance index (LCI) measurement. |
| Healthy Subjects | – | Drug: XenonUp to 50 age and sex matched control subjects (i.e., subjects with no known cardiopulmonary disorders) may also be recruited to provide a reference data set from healthy subjects for comparison. |
Who may qualify
- Ages
- 5 to 100 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- CF and Non-CF Bronchiectasis patients
Key inclusion criteria (5 listed)
- CF Patients: Diagnosis of CF based on sweat chloride >60 mMol/l
- Presence of two disease causing CFTR mutations, or end organ manifestations of disease.
- Age minimum 5 years.
- Care provided by the CCHMC CF Care Center or other regional CF Care Centers if required to achieve recruitment goals.
- Subjects 5 years of age and older with no known history of cardiopulmonary disease.
Key exclusion criteria (4 listed)
- Patients meeting standard MRI exclusions criteria (non-MRI-compatible metal implants, claustrophobia, etc.)
- Pregnancy or lactation.
- Patients meeting standard MRI exclusions criteria (non-MRI-compatible metal implants, claustrophobia, etc.)
- Pregnancy or lactation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| XenonAlso: 129XeSubjects will be scanned on a commercial 3T whole-body MRI scanner equipped with high-performance gradient systems. Natural isotopic abundance or isotopically-enriched xenon gas (~86% 129Xe, Linde Inc.) will be used for all studies. Subjects will be positioned supine in the scanner with a 129Xe RF coil around their chests. Using conventional proton MRI and a breath-hold acquisition, "scout" images will be obtained to localize the subject for subsequent 129Xe acquisitions. 129Xe images will be acquired using the same breath-hold maneuver and a pulse sequence, covering the entire lung (acquisition time <10 s, approximately 3-mm in-plane resolution, 15-mm slice thickness or less). Additional scans (sequences) may be performed. A maximum of 4 doses of 129Xe will be given throughout the study following the calibration dose. | Drug | CF and Non-CF Bronchiectasis, Healthy Subjects |
Primary outcome
Correlation of ventilated volume predictions obtained with 129Xe MRI vs 1H MRI
Time frame: Annually for 5 years
Quantify the measurement agreement between the ventilated volume of CF and Non-CF Bronchiectasis lungs vs healthy lungs
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
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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.
Zackary I Cleveland, PhD principal investigator
CCHMC
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 Cystic Fibrosis and Non-CF Bronchiectasis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
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 5 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 7 Oct 2026. 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 United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Penny A New, BBA (513-332-7931, penny.new@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Correlation of ventilated volume predictions obtained with 129Xe MRI vs 1H MRI (time frame: Annually for 5 years). 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 7 Oct 2026.