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

NCT02848560 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 38

Last updated 2024-11-21

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Hyperpolarized Xenon

Inhaled contrast for MRI occurring at each of 4 visits.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Jason Woods, PhD · Children's Hospital Medical Center, Cincinnati

Eligibility

Min Age
6 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-03-31
Primary Completion
2025-10-31
Completion
2025-10-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02848560 on ClinicalTrials.gov