Project 3: Treatment of mucostasis and airways obstruction in CF with a novel mucolytic

NIH RePORTER · NIH · P01 · $242,214 · view on reporter.nih.gov ↗

Abstract

PROJECT SUMMARY Mucus obstruction of airways is a significant pathologic feature of CF lung disease. Available therapies are only partially effective at relieving this obstruction, and additional treatments are needed. Gel-forming mucin molecules are a major constituent of CF mucus and to a large degree determines its bio-rheological properties. The development of inhaled agents that target mucin molecules and improve the transportability of airway mucus is a rational and attractive therapeutic goal. In this Project, we propose to first test the safety of a novel inhaled mucolytic in CF subjects in a single ascending dose study. We will then evaluate the ability of this agent to improve mucus clearance and reduce airways obstruction during two weeks of use, in a placebo controlled cross-over study in CF. Relevant, novel outcome measures will be utilized to evaluate drug effects on mucus clearance (gamma scintigraphy) and airways obstruction (19F-MRI with 1H-MRI/UTE), in addition to traditional clinical trial endpoints. The combination of these functional and structural modalities should greatly enhance our understanding of drug effects on this heterogeneous lung disease.

Key facts

NIH application ID
10232376
Project number
5P01HL108808-10
Recipient
UNIV OF NORTH CAROLINA CHAPEL HILL
Principal Investigator
SCOTT H DONALDSON
Activity code
P01
Funding institute
NIH
Fiscal year
2021
Award amount
$242,214
Award type
5
Project period
2017-09-07 → 2024-06-30