| Clinical trials identifier | NCT01883531 |
|---|---|
| Study codes | DPM-CF-204 |
| Study 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 |
| Sponsor | Pharmaxis |
| Drug (brand name) | Mannitol (Bronchitol) |
| Drug administration | Inhalation |
| Eligible CF patients | Children with CF aged 7 to 17 years |
| Controlled study? | Yes, placebo controlled |
| Randomised study? | Yes |
| Blinded study? | Yes (double blinded. Neither the participant, nor the medical staff knows which group the participant is in) |
| Extension study? | No |
| Study timeframe | The study was conducted between June 2013 and October 2015 |
| Study: | This Phase 2 study was conducted in 39 sites in 8 countries. |
|---|---|
| Aim: | The aim was to determine the efficacy of inhaled dry powder mannitol in improving lung function in children with CF aged 6-17 years |
| Participants: | 95 children with CF were enrolled. There were 39 participants from 13 ECFS-CTN sites in Belgium, the Netherlands, France, Germany, Italy and the UK (Leuven, Rotterdam, Reims, Montpellier, Lyon, Debré, Frankfurt, Milan, Rome, Verona, Birmingham, London, Nottingham) |
| Treatment: | Children were randomised to 1 of two groups which differed only in the sequence of treatments. In Group 1, children were treated with 4 weeks of inhaled mannitol 400 mg twice daily, followed by 4 weeks with no treatment (washout), followed by 4 weeks of inhaled placebo 400 mg twice daily. In Group 2, children were treated with 4 weeks of inhaled placebo twice daily, followed by 4 weeks with no treatment (washout), followed by 4 weeks of inhaled mannitol 400 mg twice daily. |
| Efficacy: | The primary outcome was lung function, measured by forced expiratory volume in 1 second percent predicted (FEV1pp). Lung function was significantly better (by 3.42%) when children were taking mannitol than when they were taking placebo (p=0.004). On average, children on mannitol also expectorated 1.33 grams more sputum than children on placebo (p=0.01). |
| Safety: | The rate of adverse events and serious adverse events was similar between the mannitol and placebo groups. The most common adverse events were pulmonary exacerbation and cough. The rate of pulmonary exacerbations was about 30% lower in children taking mannitol. |
| Data source: | This preliminary summary of data provided here is from clinicaltrials.gov https://clinicaltrials.gov/ct2/show/NCT01883531 And a peer reviewed publication, available here: https://www.ncbi.nlm.nih.gov/pubmed/28258928 |