Airway Clearance Therapy on Hyperpolarized 129Xenon and MRI

• Sponsor: Children's Hospital Medical Center, Cincinnati (Other)
• Overall Status: Completed
• CT.gov ID: NCT03593434
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI) (NIH)
• Enrollment: 20
• Location: 1
• Actual Duration (Months): 41
• Patients Per Site Per Month: 0.5

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: hyperpolarized Xenon gas

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 20 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: The Effect of Airway Clearance Therapy on Hyperpolarized 129Xenon MRI Compared With Lung Clearance Index and Spirometry in Cystic Fibrosis
• Actual Study Start Date: Jul 30, 2018
• Actual Primary Completion Date: Dec 30, 2021
• Actual Study Completion Date: Dec 30, 2021

Outcome Measures

Primary Outcome Measures

1. Effect of ACT on 129Xe MRI [one-day single study visit]

   Quantify the effect of ACT on 129Xe MRI in ventilation defect percentage (%), in 20 children ages 6-21 years with CF

Secondary Outcome Measures

1. Effect of ACT on spirometry [one-day single study visit]

   Quantify the effect of ACT on spirometry in indexed values of FEV1, in percentage (%), in 20 children ages 6-21 years with CF

2. Effect of ACT on LCI [one-day single study visit]

   Quantify the effect of ACT on lung clearance index (LCI) in LCI2.5 values, in 20 children ages 6-21 years with CF

Other Outcome Measures

1. Structural changes on UTE MRI [one-day single study visit]

   Describe the structural changes on UTE MRI that occur with airway clearance and correlate these with changes in spirometry and measures of ventilation heterogeneity

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Years to 21 Years
• Sexes Eligible for Study: All

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	42229

Sponsors and Collaborators

• Children's Hospital Medical Center, Cincinnati
• National Heart, Lung, and Blood Institute (NHLBI)

Investigators

• Principal Investigator: Jason Woods, PhD, Children's Hospital Medical Center, Cincinnati

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Jason Woods, PhD, Director, Center for Pulmonary Medicine Imaging Research, Principal Investigator, Children's Hospital Medical Center, Cincinnati
• ClinicalTrials.gov Identifier: NCT03593434
• Other Study ID Numbers: ACT and Xe MRI; 1R01HL131012-01A1
• First Posted: Jul 20, 2018
• Last Update Posted: Mar 10, 2022
• Last Verified: Mar 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Additional relevant MeSH terms: Cystic Fibrosis; Fibrosis; Xenon

Study Results

No Results Posted as of Mar 10, 2022