BEGIN Novel ImagiNG Biomarkers
Children's Hospital Medical Center, Cincinnati
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).
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…
Eligibility
- Age range
- 6–8 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative. 2. Willingness to adhere to the study-visit schedule and other protocol requirements. 3. Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday). 4. Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria: 1. Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test 2. Two well…
Interventions
- Drug129Xe
Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation.
Locations (3)
- University of Kansas Medical CenterKansas City, Kansas
- Carrie StevensCincinnati, Ohio
- University of VirginiaCharlottesville, Virginia