COMPLETED

Inhaled Mannitol on Mucociliary Clearance in Moderate to Severe Cystic Fibrosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This study will provide important mechanistic information regarding the effect of inhaled mannitol (Bronchitol) in people with cystic fibrosis (PwCF) with moderate to severe disease who are already using elexacaftor/tezacaftor/ivacaftor (E/T/I). Many patients have already discontinued hypertonic saline and other pulmonary therapies because of the profound effect of E/T/I of their symptoms and lung function. Further, because both inhaled osmotic agents (i.e., Bronchitol, hypertonic saline \[HS\]) and E/T/I are believed to exert their beneficial effects through improvements in mucociliary clearance (MCC), it is unknown if the combination of these therapies might be additive or are redundant in a population with moderate to severe disease where bronchiectasis and chronic infection persists, and where eventual decline in lung function is expected over time. This study, therefore, will be the first to determine whether "add on" therapy with inhaled mannitol is able to further accelerate MCC in E/T/I patients. These data would provide some guidance regarding the use of these approved therapies in PwCF.

Official Title

Effect of Bronchitol on Mucociliary Clearance in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Modulator Treated Patients With Cystic Fibrosis With Moderate to Severe Lung Disease

Quick Facts

Study Start:2023-03-28

Study Completion:2025-01-29

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:COMPLETED

Study ID

NCT05740618

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Able to provide informed consent * Age ≥ 18 at the time of screening * Diagnosis of cystic fibrosis (CF) * Regularly using elexacaftor/tezacaftor/ivacaftor (E/T/I) for ≥ 90 days * FEV1 between 30% and 70%, inclusive, at time of screening * Denies active smoking or vaping * Clinically stable with no significant changes in health status within the 28 days prior to and including the screening visit * Patients on cycled inhaled antibiotics will need to be either on or off their antibiotic for 7 days prior to Visit 1 and not scheduled to cycle during the 2-week treatment period until after Visit 2 * Has no other conditions that, in the opinion of the Site Investigator/Designee, would preclude informed consent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives	* Use of an investigational drug within 28 days prior to and including the screening visit * Unable or unwilling to withhold hypertonic saline (HS) for 4 weeks (2 weeks prior to Visit 1 and 2 weeks between Visit 1 and Visit 2) * Unable or willing to withhold dornase alfa and bronchodilators on the morning of Visit 1 and Visit 2, until completion of study procedures * Initiation of new chronic CF pulmonary therapy (e.g. dornase alfa, azithromycin, inhaled antibiotic) within 28 days prior to and including the screening visit * No acute use of antibiotics (oral, inhaled, or intravenous) or acute use of systemic corticosteroids for respiratory tract symptoms within 28 days prior to and including the screening visit. * No chronic use of oral corticosteroids \> 10 mg of prednisone or equivalent daily * Unable to tolerate albuterol or other bronchodilator * History of intolerance to HS or inhaled mannitol * Pregnancy or breast feeding * Have had more than 2 chest computed tomography (CT) in the past year or a combination of procedures that are believed to have exposed the subject's lungs to \>150 millisievert (mSv) * History of significant hemoptysis (\>60 mL) in the last three months

Inclusion Criteria

Exclusion Criteria

* Able to provide informed consent
* Age ≥ 18 at the time of screening
* Diagnosis of cystic fibrosis (CF)
* Regularly using elexacaftor/tezacaftor/ivacaftor (E/T/I) for ≥ 90 days
* FEV1 between 30% and 70%, inclusive, at time of screening
* Denies active smoking or vaping
* Clinically stable with no significant changes in health status within the 28 days prior to and including the screening visit
* Patients on cycled inhaled antibiotics will need to be either on or off their antibiotic for 7 days prior to Visit 1 and not scheduled to cycle during the 2-week treatment period until after Visit 2
* Has no other conditions that, in the opinion of the Site Investigator/Designee, would preclude informed consent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives

* Use of an investigational drug within 28 days prior to and including the screening visit
* Unable or unwilling to withhold hypertonic saline (HS) for 4 weeks (2 weeks prior to Visit 1 and 2 weeks between Visit 1 and Visit 2)
* Unable or willing to withhold dornase alfa and bronchodilators on the morning of Visit 1 and Visit 2, until completion of study procedures
* Initiation of new chronic CF pulmonary therapy (e.g. dornase alfa, azithromycin, inhaled antibiotic) within 28 days prior to and including the screening visit
* No acute use of antibiotics (oral, inhaled, or intravenous) or acute use of systemic corticosteroids for respiratory tract symptoms within 28 days prior to and including the screening visit.
* No chronic use of oral corticosteroids \> 10 mg of prednisone or equivalent daily
* Unable to tolerate albuterol or other bronchodilator
* History of intolerance to HS or inhaled mannitol
* Pregnancy or breast feeding
* Have had more than 2 chest computed tomography (CT) in the past year or a combination of procedures that are believed to have exposed the subject's lungs to \>150 millisievert (mSv)
* History of significant hemoptysis (\>60 mL) in the last three months

Contacts and Locations

Principal Investigator

Subhashini A Sellers, MD, MSCR

PRINCIPAL_INVESTIGATOR

University of North Carolina, Chapel Hill

Study Locations (Sites)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599

United States

Collaborators and Investigators

Sponsor: University of North Carolina, Chapel Hill

Subhashini A Sellers, MD, MSCR, PRINCIPAL_INVESTIGATOR, University of North Carolina, Chapel Hill

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-03-28

Study Completion Date2025-01-29

Study Record Updates

Study Start Date2023-03-28

Study Completion Date2025-01-29

Terms related to this study

Additional Relevant MeSH Terms

Cystic Fibrosis