| Clinical trials identifier | NCT02134353 |
|---|---|
| Study codes | DPM-CF-303 |
| Study title | Long Term Administration of Inhaled Mannitol in Cystic Fibrosis - A Safety and Efficacy Trial in Adult Cystic Fibrosis Subjects |
| Sponsor | Pharmaxis |
| Drug (brand name) | Mannitol (Bronchitol) |
| Drug administration | Inhalation |
| Eligible CF patients | Adults with CF |
| 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 October 2014 and September 2017. |
| Study: | This Phase 3 study was conducted in 126 sites in 21 countries. 7 CTN sites were involved (Bordeaux, Reims, Rennes, Leuven, Montpellier, Rome, Verona). |
|---|---|
| Aim: | The aim was to determine the safety and efficacy of long-term treatment with inhaled dry powder mannitol in improving lung function in adults. This study was conducted to get marketing authorisation in the US. |
| Participants: | 423 adults with CF were enrolled. |
| Treatment: | Adults were randomised to receive treatment with inhaled mannitol (400 mg twice daily) or placebo (twice daily) for 26 weeks. Mannitol has been on the market in Europe since 2012. |
| Efficacy: | Lung function, measured by forced expiratory volume in 1 second (FEV1). The change in lung function after 26 weeks of treatment was 2.24% better with mannitol than with placebo (p=0.025). The mannitol and placebo groups had similar times to pulmonary exacerbation and hospitalisations due to pulmonary exacerbation. |
| Safety: | Adverse events were similar in the mannitol and placebo groups. Overall safety was acceptable and similar to previous studies. |
| Data source: | This preliminary summary of data provided here is from a press release from the manufacturer: http://www.pharmaxis.com.au/assets/Documents/pdf/02017/ASX/2017-06-13-CF-303-TLR.pdf And from https://clinicaltrials.gov/ct2/show/study/NCT02134353?show_locs=Y |