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

Plain-language summary

This study investigates using inhaled hyperpolarized xenon MRI to monitor the response to Orkambi treatment in cystic fibrosis lung disease. It includes children aged 6 to 12 who carry two copies of the F508del CFTR mutation, or two non-functional CFTR mutations with one of them being F508del. It is a non-phase study and is currently active but not recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Children's Hospital Medical Center, Cincinnati

Intervention

Hyperpolarized Xenon

Start date

3/1/2016

Estimated completion

10/31/2025

Countries

United States

Related genes

Locations

• Cincinnati, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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