Cystic FibrosisEarly Phase 1NCT03489590
19F MRI to Evaluate Regional Ventilation in Healthy Subjects and Subjects With Cystic Fibrosis
A Early Phase 1 study testing PFP in people with Cystic Fibrosis, run by University of North Carolina, Chapel Hill at 1 site in United States.
Trial summary
An open label non-randomized study enrolling up to 20 healthy participants and up to 30 participants with cystic fibrosis to establish a healthy versus disease comparison. Each participant will receive a mixture of inert gas (perfluoropropane (PFP)) in a ratio of 79% PFP to 21% oxygen as a contrast agent to enhance… Read more about this study
- Who is it for?
- Ages 18 to 99 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- PFP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jul 2015to 31 Dec 2018
- Phase
- Early Phase 1
- 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 18 to 99 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Non-smokers (<10 pack-year history, and no active smoking in last 1 year);
- No evidence of prior lung disease or lung injury by medical history, physical exam, and/or spirometry testing;
- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the trial.
- Participants must be willing and able to comply with scheduled visits and other trial procedures.
- Non-smokers (<10 pack year history and no active smoking in the past year;
- Diagnosis of cystic fibrosis as via standard sweat chloride/phenotypic features/genotyping
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- Stable lung disease as evidenced by no change in respiratory medications or change in FEV1 of >15% from baseline over the preceding 4 weeks prior to enrollment
- Baseline FEV1 >30% of predicted. Roughly equivalent numbers of CF subjects with mild FEV1 >80% of predicted), moderate (FEV1 50-80% of predicted) and severe (FEV1 <50% of predicted) will be targeted for enrollment.
- No use of supplemental oxygen
- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the trial.
- Participants must be willing and able to comply with scheduled visits and other trial procedures.
Who cannot join
- Active or past smokers with less than 1 years since quitting or >10 pack-year smoking history
- Unable to undergo a 3.0-Tesla MRI exam of the lungs and chest because of contraindications including
- Occupation (past or present) of machiner, welder, grinder;
- Injury to the eye involving a metallic object
- Injury to the body by a metallic object (bullet, BB, shrapnel)
- Presence of a cardiac pacemaker or defibrillator
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- Presence of aneurysm clips
- Presence of carotid artery vascular clamp
- Presence of neurostimulator
- Presence of insulin or infusion pump
- Presence of implanted drug infusion device that is not known to be MRI compatible (i.e., was placed outside of UNCH or is older than 10 years)
- Bone growth or fusion simulator
- Presence of cochlear, otologic or ear implant
- Any type of prosthesis (eye, penile, etc.)
- Artificial limb or joint
- Non-removable electrodes (on body, head or brain)
- Intravascular stents, filters or coils
- Shunt (spinal or intraventricular)
- Swan-ganz catheter
- Any implant held in place by a magnet
- Transdermal delivery system (e.g. Nitro)
- IUD or diaphragm
- Tattooed makeup (eyeliner, lips, etc.) or tattoos covering >25% of body surface area
- Body piercings (MUST BE REMOVED BEFORE MRI)
- Any metal fragments
- Internal pacing wires
- Metal or wire mesh implants
- Hearing aid (REMOVE BEFORE MRI) aa. Dentures (REMOVE BEFORE MRI) bb. Claustrophobia
- Unable to tolerate inhalation of gas mixture
- Participation in a clinical trial with a study drug that may impact lung function in the past 14 days
- Other severe acute or chronic medical or psychiatric condition or clinical laboratory abnormality that may increase the risk associated with trial participation or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the participant inappropriate for entry into this trial.
- Pregnancy: women of childbearing potential must have a confirmed negative urine pregnancy test on the day of the MR scan, prior to the MRI scan.
- Active or past smokers with less than 1 years since quitting or >10 pack-year smoking history
- Active asthma flare, as perceived by the study physician or unstable asthma characterized by advancement of asthma therapy in the last month or two courses of oral steroids in the past six months.
- Unable to undergo a 3.0-Tesla MRI exam of the lungs and chest because of contraindications including
- Occupation (past or present) of machiner, welder, grinder;
- Injury to the eye involving a metallic object
- Injury to the body by a metallic object (bullet, BB, shrapnel)
- Presence of a cardiac pacemaker or defibrillator
- Presence of aneurysm clips
- Presence of carotid artery vascular clamp
- Presence of neurostimulator
- Presence of insulin or infusion pump
- Presence of implanted drug infusion device that is not known to be MRI compatible (i.e., was placed outside of UNCH or is older than 10 years)
- Bone growth or fusion simulator
- Presence of cochlear, otologic or ear implant
- Any type of prosthesis (eye, penile, etc.)
- Artificial limb or joint
- Non-removable electrodes (on body, head or brain)
- Intravascular stents, filters or coils
- Shunt (spinal or intraventricular)
- Swan-ganz catheter
- Any implant held in place by a magnet
- Transdermal delivery system (e.g. Nitro)
- IUD or diaphragm
- Tattooed makeup (eyeliner, lips, etc.) or tattoos covering >25% of body surface area
- Body piercings (MUST BE REMOVED BEFORE MRI)
- Any metal fragments
- Internal pacing wires
- Metal or wire mesh implants
- Hearing aid (REMOVE BEFORE MRI) aa. Dentures (REMOVE BEFORE MRI) bb. Claustrophobia
- Unable to tolerate inhalation of gas mixture
- Participation in a clinical trial with a study drug that may impact lung function in the past 14 days
- Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the participant inappropriate for entry into this trial.
- Pregnancy; women of childbearing potential must have a confirmed negative urine pregnancy test on the day of the MR scan, prior to the MRI scan.
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
About this study
Official title: 19F Magnetic Resonance Imaging to Evaluate Regional Ventilation in Healthy Subjects and Subjects With Cystic Fibrosis
An open label non-randomized study enrolling up to 20 healthy participants and up to 30 participants with cystic fibrosis to establish a healthy versus disease comparison. Each participant will receive a mixture of inert gas (perfluoropropane (PFP)) in a ratio of 79% PFP to 21% oxygen as a contrast agent to enhance visualization of the airway and alveolar spaces using magnetic resonance imaging of inert gas/oxygen mixtures. The study consists of a screening visit followed by up to 2 study visits.
Read the detailed description
The goal of this study is to evaluate the ability of conventional 'thermally' polarized perfluorinated gases (19F) mixed with oxygen to detect changes in ventilation using magnetic resonance imaging (MRI). A secondary goal is to assess the repeatability and the within-subject variability of these findings in CF lung disease. This is an open label pilot study expanding on work by other investigators currently using this technique in human subjects. Projection images using controlled breathing techniques will be obtained using 19F MRI, correlating spirometric variables with regional distribution of the gases.
- Conditions
- Cystic Fibrosis
- Being tested
- PFP
- Keywords
- Lung, Imaging, Hyperpolarized, Perfluorinated, MRI
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PFPDrug
Also called Perfluoropropane gas (C3F8); 19F
- Composition: inhaled PFP, a gaseous contrast agent (79% PFP, 21% O2, pre-mixed, medical grade gas)
- Administration: Full-face disposable ventilation mask and a standard Douglas Bag system.
- Dosage: Two controlled breaths of contrast gas followed by a full breath and a 12-second breath-hold and scan for image acquisition.
- Frequency: Repeated 5 times followed by an identical five cycles with room air to ensure PFP gas wash-out has occurred.
| Study group | Role | What they receive |
|---|---|---|
| 19F MRI with PFP | Experimental | PFP gas will be administered using a full-face mask during the MRI. Images are acquired during 12-second breath-hold after every 3rd breath. Before and after the MRI is complete, participants will perform spirometry maneuvers in a room outside of the magnet. Receives: Drug: PFP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Obtain technically sufficient 19F MRI images
Determine if 19F gas images of the human lung and airways can be obtained in both single breath-hold and gated breathing imaging studies with adequate signal level and image contrast to extract regional lung ventilation information.
Measured: 1 day
Secondary outcomes (2)
Determine the reproducibility of ventilation assessments in the adult cystic fibrosis (CF) population.
Repeated scans will be optional for CF participants in order to begin to understand the repeatability of this outcome measure
Measured: 1 week
Compare quantitative and qualitative image measures of lung ventilation using 19F MRI imaging to spirometric values.
Pair 19F lung images and Ventilation Defect Percentages with FEV1 obtained via spirometry
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2018
- Study start1 Jul 2015
- Primary completion31 Dec 2018
- Study completion31 Dec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of North Carolina, Chapel HillThe 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This study enrolls both healthy volunteers and volunteers with cystic fibrosis. Both healthy and CF participants will receive the same treatment throughout the entirety of the study.
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of North Carolina, Chapel Hill
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jennifer L Goralski, MD, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03489590
- Sponsor's protocol ID
- 14-0509
- Status last verified
- Oct 2019
- First submitted
- 29 Mar 2018
- Last updated
- 4 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Couch MJ, Ball IK, Li T, Fox MS, Littlefield SL, Biman B, Albert MS. Pulmonary ultrashort echo time 19F MR imaging with inhaled fluorinated gas mixtures in healthy volunteers: feasibility. Radiology. 2013 Dec;269(3):903-9. doi: 10.1148/radiol.13130609. Epub 2013 Oct 28.
PubMed 23985278Halaweish AF, Moon RE, Foster WM, Soher BJ, McAdams HP, MacFall JR, Ainslie MD, MacIntyre NR, Charles HC. Perfluoropropane gas as a magnetic resonance lung imaging contrast agent in humans. Chest. 2013 Oct;144(4):1300-1310. doi: 10.1378/chest.12-2597.
PubMed 23722696Goralski JL, Chung SH, Glass TM, Ceppe AS, Akinnagbe-Zusterzeel EO, Trimble AT, Boucher RC, Soher BJ, Charles HC, Donaldson SH, Lee YZ. Dynamic perfluorinated gas MRI reveals abnormal ventilation despite normal FEV1 in cystic fibrosis. JCI Insight. 2020 Jan 30;5(2):e133400. doi: 10.1172/jci.insight.133400.
PubMed 31855577
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 19F Magnetic Resonance Imaging to Evaluate Regional Ventilation in Healthy Subjects and Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03489590
- Acronym / sponsor ID
- 14-0509
- Conditions
- Cystic Fibrosis
- Keywords
- Lung, Imaging, Hyperpolarized, Perfluorinated, MRI
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- University of North Carolina, Chapel HillUniversity, hospital or other
- Study start
- 1 Jul 2015 (actual)
- Primary completion
- 31 Dec 2018 (actual)
- Study completion
- 31 Dec 2018 (actual)
- First posted
- 5 Apr 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.
An open label non-randomized study enrolling up to 20 healthy participants and up to 30 participants with cystic fibrosis to establish a healthy versus disease comparison. Each participant will receive a mixture of inert gas (perfluoropropane (PFP)) in a ratio of 79% PFP to 21% oxygen as a contrast agent to enhance visualization of the airway and alveolar spaces using magnetic resonance imaging of inert gas/oxygen mixtures. The study consists of a screening visit followed by up to 2 study visits.
Detailed description
The goal of this study is to evaluate the ability of conventional 'thermally' polarized perfluorinated gases (19F) mixed with oxygen to detect changes in ventilation using magnetic resonance imaging (MRI). A secondary goal is to assess the repeatability and the within-subject variability of these findings in CF lung disease. This is an open label pilot study expanding on work by other investigators currently using this technique in human subjects. Projection images using controlled breathing techniques will be obtained using 19F MRI, correlating spirometric variables with regional distribution of the gases.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This study enrolls both healthy volunteers and volunteers with cystic fibrosis. Both healthy and CF participants will receive the same treatment throughout the entirety of the study.
| Arm | Type | Receives |
|---|---|---|
| 19F MRI with PFP | Experimental | Drug: PFPPFP gas will be administered using a full-face mask during the MRI. Images are acquired during 12-second breath-hold after every 3rd breath. Before and after the MRI is complete, participants will perform spirometry maneuvers in a room outside of the magnet. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Non-smokers (<10 pack-year history, and no active smoking in last 1 year);
- No evidence of prior lung disease or lung injury by medical history, physical exam, and/or spirometry testing;
- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the trial.
- Participants must be willing and able to comply with scheduled visits and other trial procedures.
- Non-smokers (<10 pack year history and no active smoking in the past year;
Key exclusion criteria (65 listed)
- Active or past smokers with less than 1 years since quitting or >10 pack-year smoking history
- Unable to undergo a 3.0-Tesla MRI exam of the lungs and chest because of contraindications including
- Unable to tolerate inhalation of gas mixture
- Participation in a clinical trial with a study drug that may impact lung function in the past 14 days
- Other severe acute or chronic medical or psychiatric condition or clinical laboratory abnormality that may increase the risk associated with trial participation or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the participant inappropriate for entry into this trial.
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 |
|---|---|---|
| PFPAlso: Perfluoropropane gas (C3F8); 19F* Composition: inhaled PFP, a gaseous contrast agent (79% PFP, 21% O2, pre-mixed, medical grade gas) * Administration: Full-face disposable ventilation mask and a standard Douglas Bag system. * Dosage: Two controlled breaths of contrast gas followed by a full breath and a 12-second breath-hold and scan for image acquisition. * Frequency: Repeated 5 times followed by an identical five cycles with room air to ensure PFP gas wash-out has occurred. | Drug | 19F MRI with PFP |
Primary outcome
Obtain technically sufficient 19F MRI images
Time frame: 1 day
Determine if 19F gas images of the human lung and airways can be obtained in both single breath-hold and gated breathing imaging studies with adequate signal level and image contrast to extract regional lung ventilation information.
2 secondary outcomes
Determine the reproducibility of ventilation assessments in the adult cystic fibrosis (CF) population.
Time frame: 1 week
Compare quantitative and qualitative image measures of lung ventilation using 19F MRI imaging to spirometric values.
Time frame: 1 day
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 4 Oct 2019.
- Background: PubMed 23985278Couch MJ, Ball IK, Li T, Fox MS, Littlefield SL, Biman B, Albert MS. Pulmonary ultrashort echo time 19F MR imaging with inhaled fluorinated gas mixtures in healthy volunteers: feasibility. Radiology. 2013 Dec;269(3):903-9. doi: 10.1148/radiol.13130609. Epub 2013 Oct 28.
- Background: PubMed 23722696Halaweish AF, Moon RE, Foster WM, Soher BJ, McAdams HP, MacFall JR, Ainslie MD, MacIntyre NR, Charles HC. Perfluoropropane gas as a magnetic resonance lung imaging contrast agent in humans. Chest. 2013 Oct;144(4):1300-1310. doi: 10.1378/chest.12-2597.
- Derived from this study: PubMed 31855577Goralski JL, Chung SH, Glass TM, Ceppe AS, Akinnagbe-Zusterzeel EO, Trimble AT, Boucher RC, Soher BJ, Charles HC, Donaldson SH, Lee YZ. Dynamic perfluorinated gas MRI reveals abnormal ventilation despite normal FEV1 in cystic fibrosis. JCI Insight. 2020 Jan 30;5(2):e133400. doi: 10.1172/jci.insight.133400.
- 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. This study is not recruiting at the moment.
Jennifer L Goralski, MD principal investigator
University of North Carolina, Chapel Hill
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 PFP in people with Cystic Fibrosis, run by University of North Carolina, Chapel Hill 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 11 inclusion and 65 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 4 Oct 2019. 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?
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?
Obtain technically sufficient 19F MRI images (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03489590, which also lists 3 linked publications.
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 4 Oct 2019.