Thursday, July 3, 2025 7/3/2025

Regional monitoring of CF lung disease after changes in mechanical airway-clearance treatment

Award Number: R01HL131012
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/01/2016
PERIOD OF PERFORMANCE END DATE: 04/30/2028

Group Awards By:

View Award Description

Regional monitoring of CF lung disease after changes in mechanical airway-clearance treatment - PROJECT SUMMARY Cystic fibrosis (CF) is a progressive disease affecting around 30,000 people in the US and is caused by a mutation in a gene affecting the CFTR protein that regulates mucus composition. In airways in the lung this leads to mucus stasis, infection, and remodeling that result in mucus plugs, poor ventilation, and progressive airway destruction. Highly-effective CFTR modulators, now available to >90% of patients, have revolutionized clinical care, with increases in pulmonary function via more effective mucociliary clearance. Burdensome maintenance therapies like airway clearance treatment (ACT) require around 2 dedicated hours per day and have been questioned by patients, families, and medical providers. In a recent CF-community survey, airway clearance was ranked as the most burdensome therapy. Traditional studies of therapy withdrawal pose some patient risk, since measurement is via relatively insensitive pulmonary function testing (PFT) and lung-function reductions can have permanent consequences. Breakthroughs in structural and hyperpolarized-gas MRI demonstrate exquisite sensitivity to CF lung disease and can be used to monitor regional and subtle changes over time, much more precisely than PFTs, and with regional specificity. MRI provides a unique opportunity to safely evaluate ACT. The overarching goal of our proposal is to determine effectiveness of ACT in the era of highly effective CFTR modulators by studying structure-function relationships via MRI in patients of varying severity who have stopped and restarted ACT. We will achieve this goal via three separate, hypothesis-driven Aims in this clinical trial: Hypothesis 1: Patients who have relatively low structural defects will have fewer ventilation defects and higher pulmonary function, and these defects will relate to frequency of ACT usage. Specific Aim 1: To perform UTE and hyperpolarized Xe MRI in 30 CF patients aged 12-21, approximately 15 of whom have self-withdrawn ACT, to regionally characterize obstructive severity and correlate regional structural lung abnormalities (via UTE FLORET MRI) to functional deficits (via Xe MRI) Hypothesis 2: Patients who have self-withdrawn ACT after initiation of effective modulators will demonstrate increases in regional ventilation after reinitiating treatment, with greater ventilation increases in patients with a higher level of lung abnormalities. Specific Aim 2: To perform a stepwise ACT re-initiation trial in fifteen 12-21 y.o. patients who have self-withdrawn airway clearance treatment (defined as ≤ 3x/week). UTE and Xe MRI, spirometry, and multiple-breath washout will be performed at baseline, after increasing treatment to 7x/week for 1 week and then 14x/week for 2 weeks, with daily logging to aid compliance and study engagement. Hypothesis 3: Patients who currently use 2x daily ACT and have few lung abnormalities on MRI can reduce to 1x daily or less, with no significant functional changes in the lung. Specific Aim 3: To perform a stepwise ACT withdrawal trial in fifteen 12-21 y.o. patients who have low MRI abnormalities and high FEV1. Patients will be studied at baseline, after decreasing ACT to 7x/wk for 1 week, and after decreasing ACT to 3x/wk for 1 week.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = $720,465 )
2025	2025	CHILDRENS HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	001	8	4/7/2025	NON-COMPETING CONTINUATION	$720,465
2025	2024	CHILDRENS HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	7	3/19/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $720,465

Issue Date FY: 2024 ( Subtotal = $784,505 )
2024	2024	CHILDRENS HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	002	7	6/25/2024	NON-COMPETING CONTINUATION	$64,040
2024	2024	CHILDRENS HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	001	7	4/27/2024	NON-COMPETING CONTINUATION	$720,465
2024	2020	CHILDRENS HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	5	3/20/2024	NON-COMPETING CONTINUATION	$0
														Subtotal = $784,505

Issue Date FY: 2023 ( Subtotal = $802,381 )
2023	2023	CHILDRENS HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	6	8/4/2023	COMPETING CONTINUATION	$802,381
														Subtotal = $802,381

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2020	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	5	3/14/2022	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $703,064 )
2020	2020	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	5	3/31/2020	NON-COMPETING CONTINUATION	$703,064
														Subtotal = $703,064

Issue Date FY: 2019 ( Subtotal = $727,709 )
2019	2019	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	4	3/19/2019	NON-COMPETING CONTINUATION	$727,709
														Subtotal = $727,709

Issue Date FY: 2018 ( Subtotal = $736,259 )
2018	2018	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	3	3/24/2018	NON-COMPETING CONTINUATION	$721,534
2018	2018	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	001	3	6/9/2018	NON-COMPETING CONTINUATION	$14,725
2018	2018	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	002	3	8/29/2018	NON-COMPETING CONTINUATION	$0
														Subtotal = $736,259

Issue Date FY: 2017 ( Subtotal = $745,633 )
2017	2017	CHILDREN'S HOSPITAL MEDICAL CENTER	3333 BURNET AVE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	2	3/22/2017	NON-COMPETING CONTINUATION	$745,633
														Subtotal = $745,633

Issue Date FY: 2016 ( Subtotal = $760,483 )
2016	2016	CHILDREN`S HOSPITAL MEDICAL CENTER	3333 BURNET AVENUE	CINCINNATI	OH	45229	HAMILTON	USA	Lung Diseases Research	000	1	4/11/2016	NEW	$760,483
														Subtotal = $760,483

Grand Total All Awards = $5,980,499

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Regional monitoring of CF lung disease after changes in mechanical airway-clearance treatment

Award Number: R01HL131012

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 05/01/2016

PERIOD OF PERFORMANCE END DATE: 04/30/2028

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer