NCT02848560Active, not recruiting
Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI
Validation of MRI as a Sensitive Tool to Longitudinally Monitor CF Lung Disease Progression and Response to CFTR Modulator Therapy in Young Children With CF
Source: ClinicalTrials.gov · Last updated 21 Nov 2024
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 38 (estimated)
- First submitted
- 26 Jul 2016
- Start date
- 1 Mar 2016
- Primary completion date
- 31 Oct 2025
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Diseases
- Cystic fibrosis
- NCT number
- NCT02848560
What you commit to
- Study type: Observational
- Planned participants: 38
- From 1 Mar 2016 to the primary completion date, 31 Oct 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- standard MRI exclusions (metal implants, claustrophobia)
Other
- FEV1 percent predicted of \<60%
- pregnancy
Who the study is looking for
- 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
Age in the registry: 6 years to 12 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Cincinnati Children's Hospital Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.