NCT02134353

A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects

Long Term Administration of Inhaled Mannitol in Cystic Fibrosis - A Safety and Efficacy Trial in Adult Cystic Fibrosis Subjects

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 423 (actual)
• Sponsor: Syntara · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

Summary

This trial aims to provide prospective evidence of the safety and efficacy of mannitol 400 mg b.i.d. in subjects aged 18 years and above. We hypothesize that inhaled mannitol 400 mg b.i.d. will increase the mean change from baseline FEV1 (mL) compared to control over the 26-week treatment period in adult subjects with cystic fibrosis. Any improvement in FEV1 is considered clinically meaningful, however, this trial has set a threshold of 80 mL for the purposes of determining an appropriate sample size for statistical power while retaining trial feasibility in an orphan disease population

Detailed description

This is a double-blind, randomized, parallel arm, controlled, multicenter, and interventional clinical trial. Potential subjects will sign the informed consent form (ICF) and be assessed for eligibility. After satisfying all inclusion \& exclusion criteria, subjects will be given a mannitol tolerance test (MTT). Those subjects that pass the MTT will be randomized to receive inhaled mannitol (400 mg b.i.d.) or control b.i.d. for a period of 26-weeks.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Inhaled mannitol	Inhaled mannitol 400 mg BD for 26 weeks
DRUG	Placebo Comparator: Arm B - Control	Placebo Comparator: Arm B - Control BD for 26 weeks

Timeline

• Start date: 2014-09-01
• Primary completion: 2017-02-01
• Completion: 2017-02-01
• First posted: 2014-05-09
• Last updated: 2020-10-28
• Results posted: 2020-09-03

Locations

100 sites across 18 countries: United States, Argentina, Australia, Belgium, Canada, Czechia, Hungary, Israel, Italy, Mexico, New Zealand, Poland, Romania, Russia, Slovakia, South Africa, Spain, Ukraine