44 Participants Needed

Imaging Biomarkers for Cystic Fibrosis

(BEGINNING Trial)

Recruiting at 3 trial locations
PD
CS
PN
Overseen ByPenny New, BS
Age: < 18
Sex: Any
Trial Phase: Phase 4
Sponsor: Children's Hospital Medical Center, Cincinnati
Must be taking: Triple-combination therapy
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must not have changed your chronic maintenance therapies in the 28 days before joining. If you are on ivacaftor therapy with certain mutations, you cannot participate.

What data supports the effectiveness of the treatment Xenon Xe-129 for cystic fibrosis?

Research shows that using Xenon Xe-129 in MRI scans can effectively detect early lung disease in cystic fibrosis patients and monitor their response to treatment. This imaging technique is sensitive in identifying ventilation issues and has shown significant improvements in lung function measurements after treatment.12345

Is hyperpolarized 129Xe MRI safe for use in humans, particularly children with cystic fibrosis?

Research indicates that hyperpolarized 129Xe MRI is being studied for its safety and tolerability in children, including those with cystic fibrosis, but detailed safety assessments are still ongoing.12678

How is the treatment 129Xe unique for cystic fibrosis?

The treatment 129Xe is unique for cystic fibrosis because it uses hyperpolarized xenon gas in MRI scans to provide detailed images of lung ventilation, allowing for early detection and monitoring of lung disease progression, which is more sensitive than traditional methods.12459

What is the purpose of this trial?

To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).

Research Team

JW

Jason Woods, PhD

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Eligibility Criteria

This trial is for children aged 6-8 with Cystic Fibrosis, who have high sweat chloride levels and two specific CFTR gene mutations. They must be able to undergo MRI scans, be clinically stable without recent respiratory infections or changes in maintenance therapies. Kids on ivacaftor therapy or with certain mutations can't join.

Inclusion Criteria

My doctor plans to treat me with a three-drug combination.
I have been diagnosed with cystic fibrosis based on symptoms and tests.
I do not have a lung infection and my condition is stable.
See 7 more

Exclusion Criteria

I am experiencing wheezing or similar symptoms currently.
I have a long-term pancreatic condition not caused by cystic fibrosis.
I have had symptoms like coughing or wheezing in the last 28 days.
See 6 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre Trikafta Treatment

Participants undergo 129Xe MRI to establish baseline measurements before starting triple-combination therapy

4 weeks
1 visit (in-person)

Post Trikafta Treatment

Participants receive triple-combination therapy and undergo 129Xe MRI to assess treatment effects

48 weeks
4 visits (in-person)

Follow-up

Participants are monitored for changes in ventilation defect percentage, pancreas volume, and other MRI measures

4 weeks
1 visit (in-person)

Treatment Details

Interventions

  • 129Xe
Trial Overview The study tests the effects of a triple-combination therapy using advanced MRI techniques (UTE and 129Xe) after its expected approval by the FDA. It aims to assess how this treatment impacts lung and abdominal health in young patients.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: Pre TrikaftaExperimental Treatment1 Intervention
129Xe MRI
Group II: Post TrikaftaExperimental Treatment1 Intervention
129Xe MRI

129Xe is already approved in United States for the following indications:

🇺🇸
Approved in United States as Xenoview for:
  • Evaluation of lung ventilation in adults and pediatric patients aged 12 years and older

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials
844
Recruited
6,566,000+

University of Virginia

Collaborator

Trials
802
Recruited
1,342,000+

University of Kansas

Collaborator

Trials
157
Recruited
332,000+

University of Iowa

Collaborator

Trials
486
Recruited
934,000+

Findings from Research

Hyperpolarized 129Xe MRI can effectively detect ventilation defects in pediatric patients with mild cystic fibrosis (CF), even when traditional measures like FEV1 show normal results, indicating its potential as a sensitive diagnostic tool.
In a study of 22 participants (11 healthy controls and 11 CF patients), the ventilation defect percentage (VDP) was significantly higher in CF patients (18.3%) compared to controls (6.4%), suggesting that 129Xe MRI may be a valuable method for monitoring lung function and disease progression in young CF patients.
Hyperpolarized 129Xe for investigation of mild cystic fibrosis lung disease in pediatric patients.Thomen, RP., Walkup, LL., Roach, DJ., et al.[2022]
In a study involving 10 participants with mild cystic fibrosis, both dynamic 19F and hyperpolarized 129Xe MRI effectively detected ventilation abnormalities, but they provided different insights into lung function.
The results showed that while both imaging techniques identified ventilation defects, they were not entirely congruent, indicating that using both methods together can give a more comprehensive view of lung ventilation status in cystic fibrosis patients.
Comparison of single breath hyperpolarized 129 Xe MRI with dynamic 19 F MRI in cystic fibrosis lung disease.McCallister, A., Chung, SH., Antonacci, M., et al.[2021]
As more patients with cystic fibrosis (CF) receive effective CFTR modulator therapy, there is a growing need for advanced imaging tools to detect early lung disease and monitor subtle changes in condition.
This review highlights both traditional imaging methods like chest radiography and CT scans, as well as emerging techniques such as proton and hyperpolarized gas MRI, which may enhance early disease detection and treatment monitoring in CF patients.
Novel imaging techniques for cystic fibrosis lung disease.Goralski, JL., Stewart, NJ., Woods, JC.[2022]

References

Hyperpolarized 129Xe for investigation of mild cystic fibrosis lung disease in pediatric patients. [2022]
Comparison of single breath hyperpolarized 129 Xe MRI with dynamic 19 F MRI in cystic fibrosis lung disease. [2021]
Novel imaging techniques for cystic fibrosis lung disease. [2022]
The assessment of short and long term changes in lung function in CF using 129Xe MRI. [2022]
Hyperpolarised 129Xe magnetic resonance imaging to monitor treatment response in children with cystic fibrosis. [2020]
Feasibility, tolerability and safety of pediatric hyperpolarized 129Xe magnetic resonance imaging in healthy volunteers and children with cystic fibrosis. [2022]
Estimation of the radiation dose from thoracic CT scans in a cystic fibrosis population. [2016]
[A critical study of technetium 99m and xenon 133 lung scintigraphy in patients with cystic fibrosis]. [2016]
Intra- and Inter-visit Repeatability of 129 Xenon Multiple-Breath Washout MRI in Children With Stable Cystic Fibrosis Lung Disease. [2023]
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