To “combat CF” and reduce bronchiectasis onset in the disease, researchers and clinicians at Queensland Children’s Medical Research Institute are currently recruiting patients for the “COMBATCF” clinical trial, “Prevention of Bronchiectasis in Infants with Cystic Fibrosis.” Along with Stephen M. Stick, MBBChir, PhD, of Telethon Kids Institute, Peter D. Sly, MMBS, MD, DSc is leading the phase 3 study involving the drug azithromycin in children six weeks to six months of age who have cystic fibrosis.
“The general aim of this project is to conduct a randomized, double-blind, placebo-controlled clinical trial of azithromycin to determine whether treatment from infancy is safe and will prevent the onset of bronchiectasis,” wrote the authors in the trial design. To meet this goal, 150 children are expected to enroll and be treated with either azithromycin or placebo.
Of primary interest is the proportion of patients who have radiologically-defined bronchiectasis at the age of three years. Children will begin treatment at three months old (if not already older than three months) and continue with treatment until the age of three years. In addition to the bronchiectasis onset metric, a host of safety and mechanistic evaluations will be obtained. Included in the additional metrics are the extent and severity of bronchiectasis, cystic fibrosis-related quality of life, time to first pulmonary exacerbation, body mass index, and infection with Pseudomonas aeruginosa, the most commonly associated bacterium with cystic fibrosis.
Due to the long treatment window, the study is not expected to be completed until December 2017. Treatment will consist of liquid azithromycin (trade name Zithromax) three times per week or liquid placebo, depending on the enrolled patients’ assigned groups.
In an unrelated study, “Effect of Azithromycin Maintenance Treatment on Infectious Exacerbations Among Patients With Non–Cystic Fibrosis Bronchiectasis: The BAT Randomized Controlled Trial,” published in JAMA, adult non-cystic fibrosis bronchiectasis patients saw a lower rate of infectious exacerbations when they used azithromycin instead of a placebo for 12 months. This study gives hope to the current clinical trial, indicating that azithromycin may be effective at preventing, or at least treating, bronchiectasis, although it is unknown how the drug will act in young patients with cystic fibrosis.