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| ID | Type | Description | Link |
|---|---|---|---|
| 5K23HL138257-03 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
| Cystic Fibrosis Foundation | OTHER |
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The purpose of this study is to look at lung ventilation in people with cystic fibrosis over time (1 year) using magnetic resonance imaging (MRI) with an inhaled contrast gas, and compare these measures to lung function assessed by spirometry and multiple breath nitrogen washout. This study also looks at how these measures change in response to a pulmonary exacerbation and treatment (if applicable). Over the span of a year, participants would be asked to complete 3-5 visits to the University of North Carolina at Chapel Hill (UNC). with each lasting up to 4 hours. If participants do not have a pulmonary exacerbation during the year they would be asked to complete 3 visits (one at enrollment, a second roughly 2 weeks later, and the third approximately a year later). If participants do experience a CF pulmonary exacerbation they would complete 5 visits (Visit 1, Visit 2, two exacerbation visits with one before treatment and the other after, and Visit 3 at one year after Visit 1). Only one exacerbation per participant will be tracked. Participants are eligible for this study if they are 18 years old or older, have Cystic Fibrosis (CF) with mild lung disease (FEV1 >/= 60%), and can undergo an MRI. There are no known benefits for participating in this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants | All subjects to receive inhaled perfluoropropane via standard Douglas bag at every visit (3-5 visits). They will breathe the gas for 5 breath hold cycles (variable volumes as lung capacity/size varies per participant). Not to exceed 30 liters inhaled at each visit as this is the max capacity of our Douglas bag. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 19F MRI | Combination Product | Inhalation of a biologically inert contrast gas, perfluoropropane, combined with 19F-tuned MRI with image acquisition at breath-hold and during tidal breathing |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Ventilation Defect Parameter (VDP) over 14 days | VDP measured at Day 1 and Day 14, expressed as a percentage of total lung volume | Day 1 to Day 14 |
| Change in VDP over 365 days | Average of VDP measured at Day 1 and Day 14; subtracted from VDP at Day 365, expressed as a percentage of total lung volume | Day 1 to Day 365 (+/- 30 days) |
| Change in FLVlongtau2 over 14 days | FLVlongtau2 measured at Day 1 and Day 14, expressed as a percentage of total lung volume | Day 1 to Day 14 |
| Change in FLVlongtau2 over 365 days | Average of FLVlongtau2 measured at Day 1 and Day 14; subtracted from FLVlongtau2 at Day 365, expressed as a percentage of total lung volume | Day 1 to Day 365 (+/- 30 days) |
| Change in VDP occurring with a protocol-defined CF pulmonary exacerbation | Difference in VDP between baseline and exacerbation visit 1, expressed as a percentage of total lung volume. | Day 1 to Day 365 (+/- 30 days) |
| Change in FLVlongtau2 occurring with a protocol-defined CF pulmonary exacerbation | Difference in FLVlongtau2 between baseline and exacerbation visit 1, expressed as a percentage of total lung volume. | Day 1 to Day 365 (+/- 30 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Change in MRI severity score over 365 days | Change in MRI severity score from baseline to Day 365. | Day 1 to Day 365 (+/- 30 days) |
| Correlation of MRI severity score with VDP at baseline. | Baseline is a mean of the VDP scores from Day 1 and Day 14 (expressed as percentage of lung). Correlation of MRI scores with VDP and FLVlongtau2 |
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Inclusion Criteria:
Exclusion Criteria:
Subjects presenting with any of the following will not be included in the trial:
Active or past smokers or vapers with less than 1 year 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
Presence of facial hair that may interfere with the fit of the mask (and unwillingness to shave prior to each MRI)
Any changes in medications that may affect CF lung disease in the past 28 days, including any experimental therapies
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 subject 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.
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Adults with cystic fibrosis and stable lung disease at the time of enrollment
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jennifer L Goralski, MD | Contact | 919-445-0331 | jennifer_goralski@med.unc.edu | |
| Margret Z Powell, BS | Contact | 984-974-2962 | margret_powell@med.unc.edu |
| Name | Affiliation | Role |
|---|---|---|
| Jennifer L Goralski, MD | UNC Chapel Hill | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Univeristy of North Carolina at Chapel Hill | Recruiting | Chapel Hill | North Carolina | 27514 | United States |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C042852 | perflutren |
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| Day 1 to Day 14 |
| Correlation of MRI severity score with FLVlongtau2 at baseline. | Baseline is a mean of the FLVlongtau2 scores from Day 1 and Day 14 (expressed as percentage of lung). Correlation of MRI scores with VDP and FLVlongtau2 | Day 1 to Day 14 |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |