Overview

Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI

Status:
Recruiting
Trial end date:
2021-12-31
Target enrollment:
0
Participant gender:
All
Summary
This is an observational study for children with Cystic Fibrosis (CF) who are eligible based on their CF gene type. One group will be called the treatment group because they have the gene type (homozygous F508del) that makes them clinically eligible through their CF care provider to begin treatment with the new FDA approved CF drug called orkambi. For the control group, children will be enrolled who have a similar CF gene type (heterozygous F508del) but are not eligible to be prescribed orkambi. The two groups will be followed for four visits over about 3 to 4 years to observe changes in the lungs. Methods to measure the changes in lung disease will include: MRI with non-FDA approved inhaled xenon gas to take detailed images of the lungs, Pulmonary Function Tests (PFT), Lung Clearance Index (LCI), Baseline CT image of the lungs if not ordered as part of usual clinical care. The first two visits will be done before starting clinical treatment with orkambi and will be a minimum of 28 days apart and up to 18 months. The third visit will be scheduled about 3 months after starting orkambi and the fourth visit about 18 months later. For the control group, the timing of visits will be similar to treatment group and visits may be scheduled around annual CF care visits.
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Treatments:
Xenon
Criteria
Inclusion Criteria:

Treatment group:

- male or female between the ages of 6 through 12 years at enrollment

- two copies of the F508del CFTR mutation

- anticipated to be a candidate for treatment with orkambi

Control group:

- male or female between the ages of 6 through 12 years at enrollment

- two non-functional CFTR mutations with one of them being F508del CFTR mutation

- not eligible for CFTR modulation therapy

Exclusion Criteria:

- FEV1 percent predicted of <60%

- standard MRI exclusions (metal implants, claustrophobia)

- pregnancy