| Clinical trials identifier | NCT01082367 |
|---|---|
| Study codes | CTBM100C2304, EARLY |
| Study title | A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years |
| Sponsor | Novartis |
| Drug (brand name) | Tobi (tobramycin) |
| Drug administration | Inhalation |
| Eligible CF patients | People with CF aged 3 months to less than 7 years, and early lower respiratory tract infection with Pseudomonas aeruginosa (Pa) |
| 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 April 2010 and June 2015 |
| Study: | This Phase 3 study was conducted in 19 sites across 10 countries. |
|---|---|
| Aim: | The aim was to determine the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis. |
| Participants: | 51 children with CF were enrolled and randomized to receive placebo (25 children) or TOBI (26 children) twice daily for 28 days. There were 4 participants from 6 ECFS-CTN sites in France, Germany and Italy. (Bordeaux, Reims, Debré, Frankfurt, Koln, Milan). |
| Treatment: | Children received inhaled placebo or TOBI twice daily for 28 days. After this, children could carry on treatment or “cross-over” treatment for another 28 days depending on whether they were still positive for PA, as follows: • Children initially in the TOBI group and positive for Pa could continue TOBI • Children initially in the TOBI group and negative for Pa could cross over to placebo • Children initially in the placebo group and positive for Pa could cross over to TOBI • Children initially in the placebo group and negative for Pa could cross over to TOBI |
| Efficacy: | At the end of the first 28-day treatment cycle, 84.6% of children who were treated with TOBI were free from Pa infection, compared to 24% of children who were treated with placebo. The difference between groups was statistically significant (p<0.001). At the end of the 91-day testing period, the percentage of children free of PA infection was: • 76% for children who took TOBI first then switched to placebo • 47.8% for children who took placebo first then switched to TOBI |
| Safety: | There were no serious adverse events in the TOBI groups in this study. The most common adverse event was cough. |
| Data source: | This preliminary summary of data provided here is from clinicaltrials.gov https://clinicaltrials.gov/ct2/show/results/NCT01082367 |