Cystic FibrosisNCT03593434
Airway Clearance Therapy on Hyperpolarized 129Xenon and MRI
An observational study of Cystic Fibrosis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. Read more about this study
- Who is it for?
- Ages 6 to 21 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- hyperpolarized Xenon gas
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jul 2018to 30 Dec 2021
- 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 21 years
- All sexes
- Child, Adult
Study population: Male or female ages 6 through 21 with CF
Who may be able to join
- Male or female between the ages of 6 through 21 years
- CFTR genotypes as specified in NHLBI study:
- Two copies of the F508del CFTR mutation, OR Two non-functional CFTR mutations with one of them being F508del CFTR mutation
- Able to perform acceptable and repeatable spirometry
Who cannot join
- FEV1 percent predicted of <60%
- standard MRI exclusions (metal implants, claustrophobia)
- pregnancy
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 42229
About this study
Official title: The Effect of Airway Clearance Therapy on Hyperpolarized 129Xenon MRI Compared With Lung Clearance Index and Spirometry in Cystic Fibrosis
This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. That existing protocol seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with a sub-set of 20 subjects opting-in to have all of the procedures performed twice, with an intervening ACT, during one of their study visits.
Read the detailed description
This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. The existing protocol is funded by the NHLBI under R01-11863962 (PIs Woods, Clancy, and hereafter referred to as "the NHLBI study" for simplicity), and seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with patients opting-in to have all of the procedures performed twice, with an intervening ACT, during one their study visits. The target enrollment is 20 subjects.
Airway clearance therapy (ACT) is used for treating cystic fibrosis (CF). It is known to impact measures of lung function, and while an increasing number of lung function measures are used in clinical trials, there has been no direct comparison of the effects of ACT across the different modalities. This study is actively investigating the relationship between different measures of lung function, comparing a spirometric measure (FEV1 percent-predicted) with measures of whole-lung ventilation (lung clearance index, or LCI) and regional assessments of both structure (with ultra-short echo time magnetic resonance imaging, or UTE MRI) and function (with hyperpolarized xenon, or 129Xe, MRI). Performing these assessments on CF patients will help to elucidate the relative sensitivity of each measure to intra-individual changes within the lung and will help guide the selection of lung function measures in future studies.
- Conditions
- Cystic Fibrosis
- Being tested
- hyperpolarized Xenon gas
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hyperpolarized Xenon gasDrug
Also called 129Xenon
This present study will utilize existing study visits for the NHLBI study, with patients opting-in to have all of the procedures performed twice, with an intervening ACT, during one their study visits.
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of ACT on 129Xe MRI
Quantify the effect of ACT on 129Xe MRI in ventilation defect percentage (%), in 20 children ages 6-21 years with CF
Measured: one-day single study visit
Secondary outcomes (2)
Effect of ACT on spirometry
Quantify the effect of ACT on spirometry in indexed values of FEV1, in percentage (%), in 20 children ages 6-21 years with CF
Measured: one-day single study visit
Effect of ACT on LCI
Quantify the effect of ACT on lung clearance index (LCI) in LCI2.5 values, in 20 children ages 6-21 years with CF
Measured: one-day single study visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2018
- Study start30 Jul 2018
- Primary completion30 Dec 2021
- Study completion30 Dec 2021
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
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Jason Woods, Children's Hospital Medical Center, Cincinnati
- Investigators
- Jason Woods, PhD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03593434
- Sponsor's protocol ID
- ACT and Xe MRI
- Other IDs
- 1R01HL131012-01A1
- Status last verified
- Mar 2022
- First submitted
- 28 Jun 2018
- Last updated
- 10 Mar 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Airway Clearance Therapy on Hyperpolarized 129Xenon MRI Compared With Lung Clearance Index and Spirometry in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03593434
- Acronym / sponsor ID
- ACT and Xe MRI
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 30 Jul 2018 (actual)
- Primary completion
- 30 Dec 2021 (actual)
- Study completion
- 30 Dec 2021 (actual)
- First posted
- 20 Jul 2018 (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 is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. That existing protocol seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with a sub-set of 20 subjects opting-in to have all of the procedures performed twice, with an intervening ACT, during one of their study visits.
Detailed description
This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. The existing protocol is funded by the NHLBI under R01-11863962 (PIs Woods, Clancy, and hereafter referred to as "the NHLBI study" for simplicity), and seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with patients opting-in to have all of the procedures performed twice, with an intervening ACT, during one their study visits. The target enrollment is 20 subjects.
Airway clearance therapy (ACT) is used for treating cystic fibrosis (CF). It is known to impact measures of lung function, and while an increasing number of lung function measures are used in clinical trials, there has been no direct comparison of the effects of ACT across the different modalities. This study is actively investigating the relationship between different measures of lung function, comparing a spirometric measure (FEV1 percent-predicted) with measures of whole-lung ventilation (lung clearance index, or LCI) and regional assessments of both structure (with ultra-short echo time magnetic resonance imaging, or UTE MRI) and function (with hyperpolarized xenon, or 129Xe, MRI). Performing these assessments on CF patients will help to elucidate the relative sensitivity of each measure to intra-individual changes within the lung and will help guide the selection of lung function measures in future studies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 to 21 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Male or female ages 6 through 21 with CF
Key inclusion criteria (4 listed)
- Male or female between the ages of 6 through 21 years
- CFTR genotypes as specified in NHLBI study:
- Two copies of the F508del CFTR mutation, OR Two non-functional CFTR mutations with one of them being F508del CFTR mutation
- Able to perform acceptable and repeatable spirometry
Key exclusion criteria (3 listed)
- FEV1 percent predicted of <60%
- standard MRI exclusions (metal implants, claustrophobia)
- pregnancy
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 |
|---|---|---|
| hyperpolarized Xenon gasAlso: 129XenonThis present study will utilize existing study visits for the NHLBI study, with patients opting-in to have all of the procedures performed twice, with an intervening ACT, during one their study visits. | Drug | – |
Primary outcome
Effect of ACT on 129Xe MRI
Time frame: one-day single study visit
Quantify the effect of ACT on 129Xe MRI in ventilation defect percentage (%), in 20 children ages 6-21 years with CF
2 secondary outcomes
Effect of ACT on spirometry
Time frame: one-day single study visit
Effect of ACT on LCI
Time frame: one-day single study visit
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 10 Mar 2022.
- Individual participant dataSharing planned: Undecided
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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.
Jason Woods, PhD principal investigator
Children's Hospital Medical Center, Cincinnati
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, 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 4 inclusion and 3 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 10 Mar 2022. 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?
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?
Effect of ACT on 129Xe MRI (time frame: one-day single study visit). 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 10 Mar 2022.