Effect of inhaled mannitol on lung function in cystic fibrosis patients
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
PHASE4 · University of North Carolina, Chapel Hill · NCT05740618
This study is testing if adding inhaled mannitol to the current treatment can help improve lung function and clear mucus better in adults with moderate to severe cystic fibrosis.
Quick facts
| Phase | PHASE4 |
|---|---|
| Study type | Interventional |
| Enrollment | 25 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University of North Carolina, Chapel Hill (other) |
| Drugs / interventions | prednisone |
| Locations | 1 site (Chapel Hill, North Carolina) |
| Trial ID | NCT05740618 on ClinicalTrials.gov |
What this trial studies
This study investigates the impact of inhaled mannitol (Bronchitol) on mucociliary clearance in adults with moderate to severe cystic fibrosis who are already receiving elexacaftor/tezacaftor/ivacaftor (E/T/I) therapy. It aims to determine whether adding mannitol to their treatment regimen can enhance mucociliary clearance, which is crucial for lung health. The study will assess patients' lung function and symptom management over a specified treatment period. This is the first study to explore the potential additive effects of mannitol in this specific patient population.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with a diagnosis of cystic fibrosis who have been using E/T/I therapy for at least 90 days.
Not a fit: Patients who are not currently using E/T/I therapy or have severe lung function impairment (FEV1 below 30%) may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new therapeutic option to improve lung function and quality of life for cystic fibrosis patients.
How similar studies have performed: While the combination of therapies is being explored, this specific approach has not been previously tested, making it a novel investigation.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion 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 Exclusion Criteria: * 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
Where this trial is running
Chapel Hill, North Carolina
- University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States (RECRUITING)
Study contacts
- Principal investigator: Subhashini A Sellers, MD, MSCR — University of North Carolina, Chapel Hill
- Study coordinator: Subhashini A Sellers, MD, MSCR
- Email: sasellers@med.unc.edu
- Phone: 919-808-8384
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Cystic Fibrosis