Crossover Trial Determining the Efficacy of Dry Powder Mannitol to Improve Lung Function in Subjects Aged 6-17 Years
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|ClinicalTrials.gov Identifier: NCT01883531|
Recruitment Status : Completed
First Posted : June 21, 2013
Last Update Posted : October 14, 2015
It is hypothesised that inhaled mannitol 400 mg b.d. will lead to a significant improvement in the absolute change in percentage of predicted FEV1 from baseline following eight-weeks of trial treatment compared to treatment with inhaled placebo b.d.
Any improvement in FEV1 is considered clinically meaningful; however, this trial has set a threshold of 3% for the purposes of determining an appropriate sample size for statistical power whilst retaining trial feasibility in an orphan disease population.
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Inhaled Mannitol Drug: Inhaled Placebo||Phase 2|
Drug Name: Dry powder mannitol for inhalation Phase: 2 Indication: Paediatric and adolescent cystic fibrosis Trial Centres: Multicentre Sponsor: Pharmaxis Limited, 20 Rodborough Road, Frenchs Forest, NSW 2086 Australia Trial Duration: 27 weeks Number of Subjects: 160 Trial Design: Randomised, multicentre, double-blind, placebo-controlled, crossover Primary Objective: To determine the effect of eight weeks of twice-daily treatment with inhaled dry powder mannitol on lung function (FEV1) in subjects with CF who are aged six to seventeen years Dosage and Administration: Trial drug is to be administered via a dry powder inhaler.
- Mannitol 400 mg b.d. for 8 weeks followed by a 8-week washout followed by placebo b.d. for 8 weeks; or
- Placebo b.d. for 8 weeks followed by a 8-week washout followed by mannitol 400 mg b.d. for 8 weeks.
- The primary and secondary efficacy analyses will be based upon a modified Grizzle model for crossover design. Absolute and relative changes from baseline in percentage of predicted FEV1 and FVC will be analysed. The absolute change in percentage of predicted lung function (FEV1 and FVC) will be the primary focus. Changes in FEF25-75 will also be analysed.
- Safety data will be analysed descriptively (listings and summary tables).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||95 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Randomised, Multicentre, Double-blind, Placebo-controlled, Crossover Trial Determining the Efficacy of Dry Powder Mannitol in Improving Lung Function in Subjects With Cystic Fibrosis Aged Six to Seventeen Years|
|Study Start Date :||June 2013|
|Actual Primary Completion Date :||October 2015|
|Actual Study Completion Date :||October 2015|
Placebo Comparator: Inhaled Placebo
Eight-week treatment period with inhaled placebo b.d.
Drug: Inhaled Placebo
The PLacebo is non respirable mannitol due to the big size particle
Other Name: Control
Active Comparator: Inhaled Mannitol
Eight-week treatment period Inhaled Mannitol 400 mg b.d.
Drug: Inhaled Mannitol
Active treatment is inhaled mannitol with a particle size of 3-4 microns
- Effect on lung function (FEV1) [ Time Frame: The absolute change from each treatment period baseline to week 8 of each treatment period in percentage of predicted FEV1. ]To determine the effect of eight weeks of twice-daily treatment with inhaled dry powder mannitol on lung function (FEV1) in subjects with CF who are aged six to seventeen years.
- Effect on FVC [ Time Frame: The absolute change from each treatment period baseline to week 8 of each treatment period in percentage of predicted FVC. ]To determine the effect of inhaled mannitol on FVC
- Effect of inhaled mannitol on FEF25-75 [ Time Frame: The absolute change from each treatment period baseline to week 8 of each treatment period in percentage of predicted FEF25-75. ]To determine the effect of inhaled mannitol on FEF25-75 (exploratory endpoint)
- Assess safety [ Time Frame: From each treatment period baseline to week 8 of each treatment period. ]Assessment of safety will be made on the basis of reviewing changes in physical examination and using adverse event data.
- Sputum weight [ Time Frame: The absolute change from each treatment period baseline to week 8 of each treatment period in sputum weight. ]To evaluate the difference in treatment induced sputum weight in subjects treated with inhaled mannitol compared with placebo
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01883531
|John Radcliffe Hospital|
|Oxford, United Kingdom, OX3 9DU|
|Principal Investigator:||Christiane De Boeck||UZ Leuven, Belgium|
|Principal Investigator:||Jeremy Hull, Dr||John Radcliffe Hospital, Oxford, UK|
|Principal Investigator:||Anne Munck, Dr||Hôpital Robert Debré, France|
|Principal Investigator:||Joachim Riethmuller, Dr||Universitats Kinderklinik Tubingen, Germany|
|Principal Investigator:||Larry Lands, MD||'Montreal Children's Hospital, Montreal, Canada|
|Principal Investigator:||Alexander Möller, MD||University Childrens Hospital Zurich|
|Principal Investigator:||Sonia Volpi, MD||Azienda Ospedaliera Universitaria Integrata Verona Italy|
|Principal Investigator:||Harm Tiddens, MD||Erasmus MC-Sophia Children's Hospital, Rotterdam, Netherlands|