A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects
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ClinicalTrials.gov Identifier: NCT02134353 |
Recruitment Status :
Completed
First Posted : May 9, 2014
Results First Posted : September 3, 2020
Last Update Posted : October 28, 2020
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Tracking Information | |||||||
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First Submitted Date ICMJE | April 16, 2014 | ||||||
First Posted Date ICMJE | May 9, 2014 | ||||||
Results First Submitted Date ICMJE | August 3, 2020 | ||||||
Results First Posted Date ICMJE | September 3, 2020 | ||||||
Last Update Posted Date | October 28, 2020 | ||||||
Actual Study Start Date ICMJE | September 2014 | ||||||
Actual Primary Completion Date | February 2017 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4). [ Time Frame: 26 weeks ] The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach.
Least square means presented are for the average change over the 6, 14, and 26 week visits.
Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).
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Original Primary Outcome Measures ICMJE |
Mean Change in FEV1 (mL) From Baseline (Visit 1) Over the 26-week Treatment Period (to Visit 4). [ Time Frame: 26 weeks ] The mean absolute change from baseline FEV1 (mL) over weeks 6, 14 and 26 will be compared between the two treatment groups with a REML based repeated measures approach
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Change History | |||||||
Current Secondary Outcome Measures ICMJE |
Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period [ Time Frame: 26 weeks ] To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control for improving lung function as measured by mean change from baseline forced vital capacity (FVC) (mL) over the 26-week treatment period in adult subjects with cystic fibrosis (CF).
The mean absolute change from baseline FVC (mL) over 26 weeks will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach.
Least square means presented are for the change from baseline averaged over the treatment period (ie the average of the changes at 6 weeks, 14 weeks and 26 weeks).
Missing values due to withdrawal for reasons related to safety or efficacy were imputed using a baseline observation carried forward approach (BOCF).
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Original Secondary Outcome Measures ICMJE |
Mean Change From Baseline FVC (mL) Over the 26-week Treatment Period [ Time Frame: 26 weeks ] To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control for improving lung function as measured by mean change from baseline FVC (mL) over the 26-week treatment period in adult subjects with CF.
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Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures |
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Descriptive Information | |||||||
Brief Title ICMJE | A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects | ||||||
Official Title ICMJE | Long Term Administration of Inhaled Mannitol in Cystic Fibrosis - A Safety and Efficacy Trial in Adult Cystic Fibrosis Subjects | ||||||
Brief 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 |
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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. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 3 | ||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Cystic Fibrosis | ||||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Flume PA, Amelina E, Daines CL, Charlton B, Leadbetter J, Guasconi A, Aitken ML. Efficacy and safety of inhaled dry-powder mannitol in adults with cystic fibrosis: An international, randomized controlled study. J Cyst Fibros. 2021 Mar 11. pii: S1569-1993(21)00046-1. doi: 10.1016/j.jcf.2021.02.011. [Epub ahead of print] | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE |
423 | ||||||
Original Estimated Enrollment ICMJE |
440 | ||||||
Actual Study Completion Date ICMJE | February 2017 | ||||||
Actual Primary Completion Date | February 2017 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 99 Years (Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | Argentina, Australia, Belgium, Canada, Czechia, Hungary, Israel, Italy, Mexico, New Zealand, Poland, Romania, Russian Federation, Slovakia, South Africa, Spain, Ukraine, United States | ||||||
Removed Location Countries | Czech Republic, France | ||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT02134353 | ||||||
Other Study ID Numbers ICMJE | DPM-CF-303 | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product | Not Provided | ||||||
IPD Sharing Statement ICMJE | Not Provided | ||||||
Responsible Party | Pharmaxis | ||||||
Study Sponsor ICMJE | Pharmaxis | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
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PRS Account | Pharmaxis | ||||||
Verification Date | October 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |