There is new research underway involving treatment of Cystic Fibrosis in young patients using the popular antibiotic Azithromycin, which is also known by one of its brand names Zithromax. Queensland Children’s Medical Research Institute has initiated a new clinical trial to ascertain the efficacy of infancy prophylactic measures in the prevention of severe complications like bronchiectasis in Cystic Fibrosis patients with the help of Azithromycin therapy.
The research institute has started patient enrollment in the double-blind, placebo-controlled, randomized study, which will be conducted on one hundred and thirty infants with a history of Cystic Fibrosis. The Phase 3 study will test the efficacy of Azithromycin therapy against the placebo (when administered to infants from 3 months to 3 years of post-natal life) in the prevention of bronchiectasis.
Cystic Fibrosis is a genetically inherited condition that affects the mucus production and clearance from pulmonary passageways. Secretion of thick mucus and abnormal airway clearance increases the risk of pulmonary infections that increases morbidity and mortality in young CF patients.
Details of the New Cystic Fibrosis Study:
The study participants will be enrolled from CF clinics in New Zealand and Australia. The study population will be divided in the intervention group and the control group. Intervention group will receive azithromycin in a dose of 10mg/kg (thrice a week), from 3 months of age to 3 years with standard CF therapy. The control group will receive an inert liquid preparation over the same period in the same frequency, with standard CF therapy.
The inclusion criteria include children from age of 6 weeks to 6 months (of either gender) without any history of major respiratory illness, surgery or history of hospitalization or respiratory support in the first three months of life.
According to the information provided by Queensland Children’s Medical Research Institute:
Primary outcome measures of the study are:
– The percentage of children who will develop radiologically-defined bronchiectasis (that will be determined by Chest CT scan (low dose volumetric) at the age of 3 years
Secondary outcome measures of the study are:
– The overall severity and extent of bronchiectasis (that will be determined by Chest CT scan (low dose volumetric) at the age of 3 years
– The quality of life (if affected by CF at all), determined by the questionnaire at the age of 3 years
– The time at which first pulmonary exacerbation occurred. This will be determined by a standardized instrument.
– The percentage of study participants who reported a pulmonary exacerbation. A standardized instrument will be used to define the exacerbation.
– Body-mass index of study participants that will be calculated at the age of 3 years from the height and weight measurements
– Percentage of study population that will be harboring Pseudomonas aeruginosa in the bronchoalveolar secretion (in the first 3 years of study). Moreover, identification of Pseudomonas aeruginosa is also labeled as a safety issue by research team.
– The age at which the infection with Pseudomonas aeruginosa will be reported (once again a safety issue)
– The research team will conduct bronchoalveolar lavage at the age of three months, one year and towards the end of the study – at 3 years of age.
– The total volume of gas trapped in lungs that will be determined by Chest CT performed at 3 years
– Emergence of infection with organisms like; non-tuberculous mycobacterium, small colony variant Staphylococcal aureus and MRSA macrolide-resistant Staphylococcus aureus.
Queensland Children’s Medical Research Institute is sponsoring and collaborating this study with Telethon Institute for Child Heath Research that is expected to be completed by December 2016.
Because bronchiectasis is largely associated with Cystic Fibrosis in children, and particularly infants, the prospect of utilizing a macrolide antibiotic like Zithromax could pave the way for a critically important treatment method that would help to protect infant patients from destruction and widening of the large airways at such a young age.