Lung Perfusion Mapping for Cystic Fibrosis

Overseen By Lisa Clark, MA

Age: < 65

Sex: Any

Trial Phase: Phase 4

Sponsor: Children's Hospital Medical Center, Cincinnati

Must be taking: Trikafta

Disqualifiers: Metal implants, Claustrophobia, Pregnancy

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new MRI method to understand lung blood flow and air movement in children with cystic fibrosis (CF). CF is a condition where thick mucus clogs the lungs, making breathing difficult. The trial aims to determine if blood flow issues in the lungs begin before traditional breathing problems appear. Participants will include children with CF who plan to start Trikafta, a CFTR modulator. Healthy children without lung problems will join as a comparison group. As a Phase 4 trial, this research focuses on understanding how the already FDA-approved and effective treatment benefits more patients.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. However, it mentions that participants should not have used acute antibiotics for 14 days before the MRI visit.

What is the safety track record for these treatments?

Research shows that both hyperpolarized Xenon 129 and CFTR modulators are generally safe. The FDA has approved hyperpolarized Xenon 129 for children aged 6 and older to assess lung function using MRI scans. Studies have found it safe and well-tolerated in children, regardless of cystic fibrosis status.

When used in a triple combination, CFTR modulators have shown better results than a placebo and other treatments. Reports indicate that the side effects resemble those of other treatments, with no unexpected issues. Current evidence considers both treatments safe for people. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about this trial because it uses cutting-edge MRI technology with hyperpolarized Xenon 129 gas to map lung perfusion in cystic fibrosis patients. This technique allows for a non-invasive, detailed look at lung function before and after starting a CFTR modulator treatment, which is a significant step forward from traditional imaging methods. By better understanding how these modulators affect lung function, the trial is paving the way for more targeted and effective treatments for cystic fibrosis.

What evidence suggests that this trial's treatments could be effective for cystic fibrosis?

Research has shown that CFTR modulators, particularly in a triple combination, greatly improve lung function, weight, and quality of life for people with cystic fibrosis (CF). In this trial, participants in the CF cohort will undergo MRI imaging before and after starting triple-combination modulator therapy. Additionally, a special type of MRI using Hyperpolarized Xenon 129 gas, administered to both CF and control cohorts, can detect breathing problems in CF patients, even when regular lung tests appear normal. Together, these treatments and imaging methods offer promising improvements for those with CF. ^{6 7 8 9 10}

Who Is on the Research Team?

Mark DiFrancesco, PhD

Principal Investigator

CCHMC

Jason Woods, PhD

Principal Investigator

CCHMC

Are You a Good Fit for This Trial?

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 am between 6 and 21 years old.

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

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

Exclusion Criteria

Exclusion both cohorts: pregnancy

Exclusion both cohorts: standard MRI exclusions (metal implants, claustrophobia)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Assessment

Initial MRI imaging and baseline measurements for CF and control groups

1 week

1 visit (in-person)

Treatment Monitoring

CF patients undergo MRI imaging and blood sample analysis before and 6 months after initiation of triple-combination modulator therapy

6 months

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Hyperpolarized Xenon 129
• Initiation of CFTR Modulator

Trial Overview 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.

How Is the Trial Designed?

Group I: Control Cohort Experimental Treatment 1 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 Cohort Experimental Treatment 2 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.

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials

844

Recruited

6,566,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11896927/

Same‐Day Repeatability and 28‐Day Reproducibility of ... Purpose: To determine the same‐day repeatability and 28‐day reproducibility of Xe MRI in children with CF.

2. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT02606487?term=Xenon+MRI&draw=2&rank=6

Hyperpolarized Xenon MRI in Cystic Fibrosis Pulmonary ... Xe-MRI has been shown to be effective in imaging of adult patients with chronic obstructive pulmonary disease (COPD) and CF [6] however, there are no published ...

3. pubs.rsna.org pubs.rsna.org/doi/10.1148/ryct.240562

Hyperpolarized 129Xe MRI and Spectroscopy: Quantitative ... Same-day repeatability and 28-day reproducibility of xenon MRI ventilation in children with cystic fibrosis in a multi-site trial. J Magn ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5274600/

Hyperpolarized 129Xe for investigation of mild cystic ... Hyperpolarized 129Xe MRI demonstrated ventilation defects in CF patients with normal FEV1 and more effectively discriminated CF from controls ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S1076633221000131

Comparison of Hyperpolarized 3He and 129Xe MR ... In this study, we compared hyperpolarized 3 He and 129 Xe images from patients with cystic fibrosis using two commonly applied magnetic resonance sequences.

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2025/214375s001lbl.pdf

XENOVIEW (xenon Xe 129 hyperpolarized), for oral inhalation The safety and effectiveness of XENOVIEW have been established in pediatric patients aged 6 years and older for use with MRI to evaluate lung ventilation. Use ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5083137/

Feasibility, tolerability and safety of pediatric hyperpolarized ... Hyperpolarized 129Xe is a safe and well-tolerated inhaled contrast agent for pulmonary MR imaging in healthy children and in children with cystic fibrosis who ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT02912637

Hyperpolarized Xenon Imaging in Patients With Cystic ... Hyperpolarised gas MRI (3He and 129Xe) has enabled novel methods of in-vivo functional lung imaging that do not rely on the use of ionising radiation.

9. pulmonologyadvisor.com pulmonologyadvisor.com/news/xenoview-approved-for-evaluation-of-lung-ventilation-in-younger-patients/

Xenoview Approved for Evaluation of Lung Ventilation in ... The FDA has expanded the approval of Xenoview (xenon Xe 129 hyperpolarized) to include use in pediatric patients aged 6 years and older.

10. mearis.cms.gov mearis.cms.gov/public/publications/ntap/NTP221017PBF9L

MEARIS™ | Publication | ntap | NTP221017PBF9L - CMS Initial U.S. XENOVIEW(TM) is prepared using an FDA approved hyperpolarization process from Xenon Xe 129 Gas Blend dose-a safe inert gas. The HP diagnostic ...