25 Participants Needed

Inhaled Mannitol for Cystic Fibrosis

SA
MP
Overseen ByMargret Powell
Age: 18+
Sex: Any
Trial Phase: Phase 4
Sponsor: University of North Carolina, Chapel Hill
Must be taking: Elexacaftor/tezacaftor/ivacaftor
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 5 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires participants to stop using hypertonic saline for 4 weeks and to withhold dornase alfa and bronchodilators on the morning of certain visits. Participants must also not start new chronic CF pulmonary therapies or use certain antibiotics and corticosteroids shortly before and during the trial.

What data supports the effectiveness of the drug inhaled mannitol for cystic fibrosis?

Research shows that inhaled mannitol helps improve lung function and clears mucus in people with cystic fibrosis. Studies found it safe and effective, with significant improvements in lung health and quality of life for both adults and children.12345

Is inhaled mannitol safe for humans?

Inhaled mannitol, also known as Bronchitol, has been shown to be safe for use in adults with cystic fibrosis. Clinical studies confirm its safety, although a small number of patients may not tolerate it well. Proper inhaler technique and clear expectations can improve tolerance and adherence.12356

How is inhaled mannitol different from other cystic fibrosis treatments?

Inhaled mannitol is unique because it is administered as a dry powder inhaler, which helps to draw water into the airways, making mucus less sticky and easier to clear. This mechanism of action is different from other treatments that may focus on breaking down mucus or reducing inflammation.7891011

Research Team

SA

Subhashini A Sellers, MD, MSCR

Principal Investigator

University of North Carolina, Chapel Hill

Eligibility Criteria

Adults over 18 with cystic fibrosis who've been on elexacaftor/tezacaftor/ivacaftor for at least 90 days, have an FEV1 between 30-70%, and are non-smokers. They must be stable health-wise and not on certain other treatments or medications that could affect the trial results.

Inclusion Criteria

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
Able to provide informed consent
I will not start or stop inhaled antibiotics 7 days before my first visit and won't change them for 2 weeks after.
See 5 more

Exclusion Criteria

Use of an investigational drug within 28 days prior to and including the screening visit
I can stop using hypertonic saline for 4 weeks.
You cannot tolerate albuterol or other medicines that help open up the airways.
See 8 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive dry powder mannitol 400 mg twice a day by oral inhalation for 14 days

2 weeks
2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Mannitol Inhalant Product
Trial Overview The study is testing if adding inhaled mannitol (Bronchitol) to patients' current regimen of elexacaftor/tezacaftor/ivacaftor can further improve mucociliary clearance in those with moderate to severe cystic fibrosis.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Inhaled MannitolExperimental Treatment1 Intervention
All study participants will receive the same study treatment. Study treatment will be dry powder mannitol 400 mg twice a day by oral inhalation (the contents of 10 capsules administered individually) for 14 days +/- 2 days.

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of North Carolina, Chapel Hill

Lead Sponsor

Trials
1,588
Recruited
4,364,000+

Chiesi USA, Inc.

Industry Sponsor

Trials
9
Recruited
6,100+

Findings from Research

Inhaled mannitol is a safe and effective treatment for adult patients with cystic fibrosis, showing significant improvements in lung function in large phase 3 studies.
Proper training in inhaler technique and setting clear expectations can enhance patient tolerance and adherence to mannitol therapy, which is crucial for long-term efficacy.
Optimising inhaled mannitol for cystic fibrosis in an adult population.Flume, PA., Aitken, ML., Bilton, D., et al.[2020]
Inhaled mannitol (Bronchitol®) is a cost-effective treatment for cystic fibrosis when added to best supportive care, with a cost of AU$39,165 per quality-adjusted life-year, making it a viable option for the Australian healthcare system.
The analysis was based on a comprehensive model using data from phase III trials and Australian CF registries, showing that 77% of simulations indicated mannitol's cost-effectiveness below the AU$45,000 threshold for life-threatening diseases.
Cost Effectiveness of Inhaled Mannitol (Bronchitol®) in Patients with Cystic Fibrosis.Warren, E., Morgan, K., Toward, TJ., et al.[2020]
In a study involving 423 adults with cystic fibrosis, inhaling 400 mg of mannitol twice daily for 26 weeks significantly improved lung function (FEV1) compared to a control group, with an average increase of 54 mL.
Mannitol was found to have a good safety profile, with most adverse events being mild or moderate, and only a slightly higher percentage of treatment-related adverse events compared to the control group.
Efficacy and safety of inhaled dry-powder mannitol in adults with cystic fibrosis: An international, randomized controlled study.Flume, PA., Amelina, E., Daines, CL., et al.[2022]

References

Optimising inhaled mannitol for cystic fibrosis in an adult population. [2020]
Cost Effectiveness of Inhaled Mannitol (Bronchitol®) in Patients with Cystic Fibrosis. [2020]
Efficacy and safety of inhaled dry-powder mannitol in adults with cystic fibrosis: An international, randomized controlled study. [2022]
Inhaled dry powder mannitol in children with cystic fibrosis: A randomised efficacy and safety trial. [2018]
Inhaled mannitol in patients with cystic fibrosis: A randomised open-label dose response trial. [2013]
Safety assessment of inhaled xylitol in subjects with cystic fibrosis. [2013]
Glycopyrrolate/formoterol fumarate metered dose inhaler for maintenance-naïve patients with chronic obstructive pulmonary disease: a post-hoc analysis of the randomized PINNACLE trials. [2020]
Real-life therapeutic effects of beclomethasone dipropionate/formoterol fumarate/glycopyrronium combined triple therapy in patients with chronic obstructive pulmonary disease. [2023]
Pharmacokinetics of glycopyrronium/formoterol fumarate dihydrate delivered via metered dose inhaler using co-suspension delivery technology in patients with moderate-to-very severe COPD. [2018]
Comparative efficacy and safety of glycopyrronium/formoterol fixed-dose combination versus glycopyrronium monotherapy in patients with moderate-to-severe COPD. [2023]
Long-term safety and efficacy of glycopyrrolate/formoterol metered dose inhaler using novel Co-Suspension™ Delivery Technology in patients with chronic obstructive pulmonary disease. [2022]
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