Clinical trial NCT03593434: Airway Clearance Therapy on Hyperpolarized 129Xenon and MRI
Official title: The Effect of Airway Clearance Therapy on Hyperpolarized 129Xenon MRI Compared With Lung Clearance Index and Spirometry in Cystic Fibrosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2018-07-20
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 21
Standard ages: CHILD
ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male or female ages 6 through 21 with CF

Brief summary: This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. That existing protocol seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with a sub-set of 20 subjects opting-in to have all of the procedures performed twice, with an intervening ACT, during one of their study visits.

Detailed description: This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. The existing protocol is funded by the NHLBI under R01-11863962 (PIs Woods, Clancy, and hereafter referred to as "the NHLBI study" for simplicity), and seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with patients opting-in to have all of the procedures performed twice, with an intervening ACT, during one their study visits. The target enrollment is 20 subjects.

Airway clearance therapy (ACT) is used for treating cystic fibrosis (CF). It is known to impact measures of lung function, and while an increasing number of lung function measures are used in clinical trials, there has been no direct comparison of the effects of ACT across the different modalities. This study is actively investigating the relationship between different measures of lung function, comparing a spirometric measure (FEV1 percent-predicted) with measures of whole-lung ventilation (lung clearance index, or LCI) and regional assessments of both structure (with ultra-short echo time magnetic resonance imaging, or UTE MRI) and function (with hyperpolarized xenon, or 129Xe, MRI). Performing these assessments on CF patients will help to elucidate the relative sensitivity of each measure to intra-individual changes within the lung and will help guide the selection of lung function measures in future studies.

Eligibility criteria: Inclusion Criteria:

* Male or female between the ages of 6 through 21 years
* CFTR genotypes as specified in NHLBI study:

Two copies of the F508del CFTR mutation, OR Two non-functional CFTR mutations with one of them being F508del CFTR mutation

• Able to perform acceptable and repeatable spirometry

Exclusion Criteria:

* FEV1 percent predicted of \<60%
* standard MRI exclusions (metal implants, claustrophobia)
* pregnancy

Primary outcomes:
measure: Effect of ACT on 129Xe MRI
timeFrame: one-day single study visit
Secondary outcomes:
measure: Effect of ACT on spirometry
timeFrame: one-day single study visit
measure: Effect of ACT on LCI
timeFrame: one-day single study visit
Other outcomes:
measure: Structural changes on UTE MRI
timeFrame: one-day single study visit
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Jason Woods, PhD
role: PRINCIPAL_INVESTIGATOR
References: