Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis

NCT06057714 | Recruiting DMC FDA Drug

• 2 other identifiers: 20-12335K23HL138257-03
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis; Ventilatory Defect

Outcome Measures

Primary Outcomes (6)

• 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)

Secondary Outcomes (3)

• Change in MRI severity score over 365 days

  Day 1 to Day 365 (+/- 30 days)

• Correlation of MRI severity score with VDP at baseline.

  Day 1 to Day 14

• Correlation of MRI severity score with FLVlongtau2 at baseline.

  Day 1 to Day 14

Study Arms (1)

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.

Combination Product: 19F MRI

Interventions

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

Also known as: Perfluoropropane (PFP)

All Participants

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults with cystic fibrosis and stable lung disease at the time of enrollment

You may qualify if:

• Subjects must be ≥18 years of age
• Non-smokers (\<10 pack/year history and no active smoking in the past year)
• Diagnosis of cystic fibrosis via standard sweat chloride/phenotypic features/genotyping
• No use of supplemental oxygen
• Stable lung function (within 10% of personal best in the last 6 months) with no pulmonary exacerbations in the past 4 weeks and baseline FEV1≥60% of predicted
• Evidence of a personally signed and dated consent indicating that the subject has been informed of all pertinent aspects of the trial
• Subjects must be willing and able to comply with scheduled visits and other trial procedures

You may not qualify if:

• 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
• Occupation (past or present) of machinist, welder, or grinder;
• Injury to the eye involving a metallic object
• Injury to the body by a metallic object (bullet, bullet ball, 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 UNC 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.)

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): collaborator
• Cystic Fibrosis Foundation: collaborator
• University of North Carolina, Chapel Hill: lead

Study Sites (1)

Univeristy of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

perflutren

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Jennifer L Goralski, MD

  UNC Chapel Hill

  PRINCIPAL INVESTIGATOR

Central Study Contacts

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

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2023
• First Posted: September 28, 2023
• Study Start: January 20, 2021
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: February 17, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)