BEGIN Novel ImagiNG Biomarkers
Start Date
5/1/2022
Completion Date
11/1/2028
Summary
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).
Detailed Description
The overall hypothesis is that multi-organ MRI will provide more sensitive, robust outcome measures in young CF patients than traditional measures employed in the BEGIN study and that these novel measures will be more sensitive to treatment effects, tested here by comparison before and after triple-combination modulator therapy. By understanding the nature of early lung obstruction and characteristic changes in the liver and pancreas over time, we continue to lay the groundwork for more personalized medicine in the future. Assessing treatment response and clinical benefit in children with CF who are clinically normal per standard outcomes (e.g., spirometry, pancreatic function) will become paramount as triplecombination therapy is extended to younger patients with milder CF clinical presentation than their historic peers. Here the sensitivity and profile free of ionizing-radiation exposure of MRI can be leveraged to follow an individual with CF over time to quantify changes with therapy-with additional spatial resolution unavailable from standard clinical testing.
Eligibility Criteria
Age Range: 6 years to 8 years
Interventions
129Xe
Conditions
Locations
University of Kansas Medical Center
Kansas City, Kansas 66160
United States
Carrie Stevens
Cincinnati, Ohio 45229
United States
University of Virginia
Charlottesville, Virginia 22903
United States