Crossover Trial Determining the Efficacy of Dry Powder Mannitol to Improve Lung Function in Subjects Aged 6-17 Years
Status:
Completed
Trial end date:
2015-10-01
Target enrollment:
Participant gender:
Summary
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.