Lung Perfusion Mapping for Cystic Fibrosis

Palo Alto (17 mi)

Overseen byMark DiFrancesco, PhD

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: Children's Hospital Medical Center, Cincinnati

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?Cystic fibrosis (CF) results in the thickening of mucus in the lungs and other organs due to dysfunction of a transmembrane conductance protein. This allows buildup of bacteria that results in inflammation, leading to tissue breakdown and loss of function. In the lungs, this process causes loss of air exchange structures progressing to diminished lung function. The exchange of oxygen in the lungs depends on both the integrity of air conduits and vasculature. Most clinical assessments, however, focus on ventilatory function, with the assumption that any vascular compromise is secondary. Nevertheless, there is evidence, some from the investigator's lab, to suggest that perfusion anomalies in the lung occur before signs of ventilatory dysfunction. Thus, the inflammatory processes of CF may impact pulmonary microvasculature specifically and concurrently or prior to damage to ventilatory structures. This study aims to apply a new MRI method to serially measure regional lung perfusion, without the use of contrast agent, in children with CF and to associate it with regional assessments of ventilation and to serum cytokines or proteomic markers of angiogenesis and inflammatory processes. The investigator's lab has recently developed a noninvasive, non-contrast, method of labeling blood flowing into the lungs and generating a map of perfusion. The investigator aims to couple this technique to existing methods using hyperpolarized Xenon to map ventilation. The investigator will apply these methods over time in CF patients, monitoring the relationship between regional perfusion and ventilation defects. This pilot work will provide the foundation for larger studies to establish the essential etiological role of perfusion deficits in CF.

Eligibility Criteria

This trial is for children and young adults aged 6-21 with cystic fibrosis (CF) confirmed by tests, who are about to start Trikafta treatment. Healthy individuals of the same age without lung issues can also join as controls. Participants need stable lung function and no recent exacerbations or antibiotic treatments.

Inclusion Criteria

I have been diagnosed with cystic fibrosis through a sweat and genetic test.

My oxygen levels stay above 90% when I'm lying down.

I am between 6 and 21 years old and healthy.

My lung function is within 5% of my best result in the past 6 months.

Treatment Details

The study tests a new MRI technique that maps blood flow in the lungs without contrast agents, alongside hyperpolarized Xenon to map ventilation. It aims to understand how CF affects lung perfusion and its relationship with ventilation defects over time.

2Treatment groups

Experimental Treatment

Group I: Control CohortExperimental Treatment1 Intervention

10 Healthy control study participants matched for age and gender will undergo one MRI imaging study visit. Hyperpolarized Xenon 129 will be administered through inhalation at one MRI imaging study visit.

Group II: CF CohortExperimental Treatment2 Interventions

16 Cystic Fibrosis Patients will undergo MRI imaging before and 6 months after initiation of triple-combination modulator therapy. Initiation of triple -combination modulator therapy will be determined by clinician and family prior to study enrollment. Hyperpolarized Xenon 129 will be administered through inhalation at two MRI imaging study visits.