Cystic FibrosisEarly Phase 1NCT06339593
Regional Monitoring of CF Lung Disease
A Early Phase 1 study testing Airway-clearance vest and Hyperpolarized Xe129 in people with Cystic Fibrosis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The main reason for this research study is to learn more about some new tests that are being developing for patients with Cystic Fibrosis (CF) to measure changes in the lungs. In this study, the focus will be to learn how stopping Airway Clearance (ACT) and re-starting ACT can affect these tests. Read more about this study
- Who is it for?
- Ages 12 to 21 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Airway-clearance vest and Hyperpolarized Xe129
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jan 2024to Jan 2028
- Phase
- Early Phase 1
- 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 12 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1 Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
- 2 Use of highly effective modulators for more than 30 days (ie. Trikafta) 3 Willingness and ability to adhere to the study visit schedule and other protocol requirements.
- 4 Documentation of a CF diagnosis with prescription of Mechanical ACT 5 Ages 12-21 inclusive, at the time of consent. 6 Clinically stable with no respiratory tract infection or recent exacerbations. 7 Treating CF physician agreeable to study procedures. Only applicable to Aim 3.
- 8 No change in chronic maintenance therapies in the 28 days prior to enrollment.
- 9 Ability to cooperate with MRI procedures.
Who cannot join
- Standard MRI exclusions (metal implants, claustrophobia).
- For females of childbearing potential: Positive urine pregnancy test or Lactating.
- Acute respiratory symptoms (e.g., wheezing) at the time of the MRI
- Chronic lung or liver or pancreatic disease not related to CF.
- Any other condition that, in the opinion of the Investigator, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
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
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Carrie Stevens, BS, (513) 636-9973, carrie.stevens@cchmc.org
About this study
Official title: Regional Monitoring of CF Lung Disease After Changes in Mechanical Airway-clearance Treatment
The main reason for this research study is to learn more about some new tests that are being developing for patients with Cystic Fibrosis (CF) to measure changes in the lungs. In this study, the focus will be to learn how stopping Airway Clearance (ACT) and re-starting ACT can affect these tests. These new tests include using a breathable gas called Xenon (Xe) with MRI (magnetic resonance imaging) to improve the pictures of changes in the lungs. The Xenon (Xe) gas that has been treated to have a larger MRI signal (also called hyperpolarized). The other new test is called LCI (Lung Clearance Index) that can measure how well the lungs are working. The MRI machine used in this study has been approved by the U.S. Food and Drug Administration (FDA) and is commercially available for sale in the USA. Hyperpolarized Xe gas is an FDA-approved, inhaled contrast agent for lung ventilation MRI. The new Xe MRI techniques that are being developed and used for this research study are investigational, meaning these new Xe MRI techniques are not FDA approved, but they are similar to FDA-approved techniques that are used clinically at Cincinnati Children's Hospital Medical Center (CCHMC). Xe gas and the new MRI techniques used in this research study have been used for many years in research, including in many research studies conducted at CCHMC like this one.
Read the detailed description
Cystic fibrosis (CF) is a progressive, systemic disease affecting an estimated 30,000 children and adults in the United States (70,000+ worldwide) and is caused by mutations in the gene that encodes the cystic fibrosis transmembrane conductance regulator (CFTR) protein--a chloride and bicarbonate channel that regulates ion transport and mucus composition in CF-affected tissues, such as the lung. In airways this leads to mucus stasis, infection, inflammation, and remodeling that result in mucus plugs, regional lung obstruction, and progressive airway destruction and bronchiectasis. Highly-effective CFTR modulators, which are recently available to >90% of patients, have revolutionized CF clinical care, with large increases in pulmonary function as a result of more effective mucociliary clearance. As a result, burdensome maintenance therapies like mechanical airway clearance treatment (ACT), requiring nearly 2 dedicated hours per day, have been questioned by patients, families, and medical providers. In a recent survey of CF community members, ACT was ranked as the most burdensome chronic therapy, yet is the least studied. Prospective studies of maintenance-therapy withdrawal pose potential ethical risks, since traditional testing via spirometry and/or multiple-breath washout is relatively insensitive to small or regional changes and long-term lung-function reductions often have permanent consequences. Nevertheless, many patients have withdrawn these maintenance therapies against advice from their providers. A major gap in CF management is our ability to monitor lung function sensitively and rapidly as a result of treatment changes, such as partial withdrawal of ACT.
Breakthroughs in structural and functional magnetic resonance imaging (MRI) have demonstrated exquisite sensitivity to regional CF lung disease and can monitor regional and subtle changes over time, without ionizing radiation, even in patients with normal spirometry. As demonstrated in the previous R01 that ultrashort echo time (UTE) MRI provides structural images that rival computered tomography (CT) imaging, with sensitivity to detect all of the structural hallmarks of treatable (e.g., mucus plugs) and permanent lung disease (e.g., bronchiectasis). It has been demonstrated that hyperpolarized 129Xe MRI is more sensitive than any other technique at detecting changes in regional pulmonary ventilation and gas exchange. For the first time, a single modality (MRI) is available to safely monitor regional lung disease and treatment changes before FEV1 declines become permanent. This is a unique opportunity to safely evaluate ACT in CF populations that remain at risk of long-term lung function decline.
- Conditions
- Cystic Fibrosis
- Being tested
- Airway-clearance vest, Hyperpolarized Xe129
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Airway-clearance vestProcedure or surgery
Airway clearance devices will be re-initiated for those who have self withdrawn and will be withdrawn temporarily for those who are using them regularly and have high FEV1 and low Xe MRI ventilation defects.
Hyperpolarized Xe129Drug
Hyperpolarized Xe MRI is FDA Approved for evaluation of ventilation in adults and children 12 and above and will be used as a diagnostic test here. Earlier studies using Xe MRI have shown its efficacy in exploring CF and other lung diseases in pediatric and adult populations, and it has been found to be much more sensitive to early lung disease than traditional metrics like spirometry and MBW.
| Study group | Role | What they receive |
|---|---|---|
| CF pts on MACT | Other | Males and females from 12 to 21 years old who have been prescribed Mechanical Airway Clearance Therapy for CF. Receives: Drug: Hyperpolarized Xe129 |
| Self withdrawn ACT | Other | To perform a stepwise ACT re-initiation trial in fifteen 12-21 y.o. patients who have self-withdrawn airway clearance treatment (defined as ≤ 3x/week). UTE and Xe MRI, spirometry, and multiple-breath washout will be performed at baseline, after increasing treatment to 7x/week for 1 week and then 14x/week for 2 weeks, with daily logging to aid compliance and study engagement. Receives: Procedure: Airway-clearance vest; Drug: Hyperpolarized Xe129 |
| Low MRI abnormalities and high FEV1 | Other | To perform a stepwise ACT withdrawal trial in fifteen 12-21 y.o. patients who have low MRI abnormalities and high FEV1. Patients will be studied at baseline, after decreasing ACT to 7x/week for 1 week, and after decreasing ACT to 3x/week for 1 week. Receives: Procedure: Airway-clearance vest; Drug: Hyperpolarized Xe129 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessing functional lung abnormalities via Xenon MRI
We will be using Hyperpolarized Xenon MRI images of participants with Cystic Fibrosis
Measured: two weeks
Secondary outcomes (3)
Assessing patients who have self-withdrawn airway clearance treatment to determine ventilation abnormalities.
UTI MRI will be used to measure participants airway clearance
Measured: two weeks
Assessing patients who have self-withdrawn airway clearance treatment to determine ventilation abnormalities.
Spirometry will be used to measure participants airway clearance
Measured: two weeks
Assessing patients who have self-withdrawn airway clearance treatment to determine
Multiple-breath washout will be used to measure participants airway clearance
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2024
- Study start5 Jan 2024
- Primary completionJan 2028 (estimated)
- Study completionJan 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
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jason Woods, Children's Hospital Medical Center, Cincinnati
- Investigators
- Jason C Woods, PhD, Principal investigator, Cincinnati Children's Hospital Medica Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06339593
- Sponsor's protocol ID
- 2023-0391
- Status last verified
- Jan 2026
- First submitted
- 6 Feb 2024
- Last updated
- 14 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Regional Monitoring of CF Lung Disease After Changes in Mechanical Airway-clearance Treatment
- ClinicalTrials.gov ID
- NCT06339593
- Acronym / sponsor ID
- 2023-0391
- Conditions
- Cystic Fibrosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 5 Jan 2024 (actual)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 1 Apr 2024 (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 main reason for this research study is to learn more about some new tests that are being developing for patients with Cystic Fibrosis (CF) to measure changes in the lungs. In this study, the focus will be to learn how stopping Airway Clearance (ACT) and re-starting ACT can affect these tests. These new tests include using a breathable gas called Xenon (Xe) with MRI (magnetic resonance imaging) to improve the pictures of changes in the lungs. The Xenon (Xe) gas that has been treated to have a larger MRI signal (also called hyperpolarized). The other new test is called LCI (Lung Clearance Index) that can measure how well the lungs are working. The MRI machine used in this study has been approved by the U.S. Food and Drug Administration (FDA) and is commercially available for sale in the USA. Hyperpolarized Xe gas is an FDA-approved, inhaled contrast agent for lung ventilation MRI. The new Xe MRI techniques that are being developed and used for this research study are investigational, meaning these new Xe MRI techniques are not FDA approved, but they are similar to FDA-approved techniques that are used clinically at Cincinnati Children's Hospital Medical Center (CCHMC). Xe gas and the new MRI techniques used in this research study have been used for many years in research, including in many research studies conducted at CCHMC like this one.
Detailed description
Cystic fibrosis (CF) is a progressive, systemic disease affecting an estimated 30,000 children and adults in the United States (70,000+ worldwide) and is caused by mutations in the gene that encodes the cystic fibrosis transmembrane conductance regulator (CFTR) protein--a chloride and bicarbonate channel that regulates ion transport and mucus composition in CF-affected tissues, such as the lung. In airways this leads to mucus stasis, infection, inflammation, and remodeling that result in mucus plugs, regional lung obstruction, and progressive airway destruction and bronchiectasis. Highly-effective CFTR modulators, which are recently available to >90% of patients, have revolutionized CF clinical care, with large increases in pulmonary function as a result of more effective mucociliary clearance. As a result, burdensome maintenance therapies like mechanical airway clearance treatment (ACT), requiring nearly 2 dedicated hours per day, have been questioned by patients, families, and medical providers. In a recent survey of CF community members, ACT was ranked as the most burdensome chronic therapy, yet is the least studied. Prospective studies of maintenance-therapy withdrawal pose potential ethical risks, since traditional testing via spirometry and/or multiple-breath washout is relatively insensitive to small or regional changes and long-term lung-function reductions often have permanent consequences. Nevertheless, many patients have withdrawn these maintenance therapies against advice from their providers. A major gap in CF management is our ability to monitor lung function sensitively and rapidly as a result of treatment changes, such as partial withdrawal of ACT.
Breakthroughs in structural and functional magnetic resonance imaging (MRI) have demonstrated exquisite sensitivity to regional CF lung disease and can monitor regional and subtle changes over time, without ionizing radiation, even in patients with normal spirometry. As demonstrated in the previous R01 that ultrashort echo time (UTE) MRI provides structural images that rival computered tomography (CT) imaging, with sensitivity to detect all of the structural hallmarks of treatable (e.g., mucus plugs) and permanent lung disease (e.g., bronchiectasis). It has been demonstrated that hyperpolarized 129Xe MRI is more sensitive than any other technique at detecting changes in regional pulmonary ventilation and gas exchange. For the first time, a single modality (MRI) is available to safely monitor regional lung disease and treatment changes before FEV1 declines become permanent. This is a unique opportunity to safely evaluate ACT in CF populations that remain at risk of long-term lung function decline.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| CF pts on MACT | Other | Drug: Hyperpolarized Xe129Males and females from 12 to 21 years old who have been prescribed Mechanical Airway Clearance Therapy for CF. |
| Self withdrawn ACT | Other | Procedure: Airway-clearance vest, Drug: Hyperpolarized Xe129To perform a stepwise ACT re-initiation trial in fifteen 12-21 y.o. patients who have self-withdrawn airway clearance treatment (defined as ≤ 3x/week). UTE and Xe MRI, spirometry, and multiple-breath washout will be performed at baseline, after increasing treatment to 7x/week for 1 week and then 14x/week for 2 weeks, with daily logging to aid compliance and study engagement. |
| Low MRI abnormalities and high FEV1 | Other | Procedure: Airway-clearance vest, Drug: Hyperpolarized Xe129To perform a stepwise ACT withdrawal trial in fifteen 12-21 y.o. patients who have low MRI abnormalities and high FEV1. Patients will be studied at baseline, after decreasing ACT to 7x/week for 1 week, and after decreasing ACT to 3x/week for 1 week. |
Who may qualify
- Ages
- 12 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 1 Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
- 2 Use of highly effective modulators for more than 30 days (ie. Trikafta) 3 Willingness and ability to adhere to the study visit schedule and other protocol requirements.
- 4 Documentation of a CF diagnosis with prescription of Mechanical ACT 5 Ages 12-21 inclusive, at the time of consent. 6 Clinically stable with no respiratory tract infection or recent exacerbations. 7 Treating CF physician agreeable to study procedures. Only applicable to Aim 3.
- 8 No change in chronic maintenance therapies in the 28 days prior to enrollment.
- 9 Ability to cooperate with MRI procedures.
Key exclusion criteria (5 listed)
- Standard MRI exclusions (metal implants, claustrophobia).
- For females of childbearing potential: Positive urine pregnancy test or Lactating.
- Acute respiratory symptoms (e.g., wheezing) at the time of the MRI
- Chronic lung or liver or pancreatic disease not related to CF.
- Any other condition that, in the opinion of the Investigator, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
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 |
|---|---|---|
| Airway-clearance vestAirway clearance devices will be re-initiated for those who have self withdrawn and will be withdrawn temporarily for those who are using them regularly and have high FEV1 and low Xe MRI ventilation defects. | Procedure or surgery | Low MRI abnormalities and high FEV1, Self withdrawn ACT |
| Hyperpolarized Xe129Hyperpolarized Xe MRI is FDA Approved for evaluation of ventilation in adults and children 12 and above and will be used as a diagnostic test here. Earlier studies using Xe MRI have shown its efficacy in exploring CF and other lung diseases in pediatric and adult populations, and it has been found to be much more sensitive to early lung disease than traditional metrics like spirometry and MBW. | Drug | CF pts on MACT, Low MRI abnormalities and high FEV1, Self withdrawn ACT |
Primary outcome
Assessing functional lung abnormalities via Xenon MRI
Time frame: two weeks
We will be using Hyperpolarized Xenon MRI images of participants with Cystic Fibrosis
3 secondary outcomes
Assessing patients who have self-withdrawn airway clearance treatment to determine ventilation abnormalities.
Time frame: two weeks
Assessing patients who have self-withdrawn airway clearance treatment to determine ventilation abnormalities.
Time frame: two weeks
Assessing patients who have self-withdrawn airway clearance treatment to determine
Time frame: 2 weeks
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 14 Jan 2026.
- Individual participant dataSharing planned: No
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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.
Jason C Woods, PhD principal investigator
Cincinnati Children's Hospital Medica Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Airway-clearance vest and Hyperpolarized Xe129 in people with Cystic Fibrosis, 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 5 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 14 Jan 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Carrie Stevens, BS ((513) 636-9973, carrie.stevens@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessing functional lung abnormalities via Xenon MRI (time frame: two weeks). 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 14 Jan 2026.