Study designs and challenges in clinical studies conducted in infants and children with asthma

Because of the challenges associated with conducting clinical trials in ped iatric patients, most drugs have not been adequately tested in this patient population. Insufficient testing frequently results in product labeling th at fails to provide instructions for safe and effective use in pediatric pa tients. The most prevalent chronic disease in children is asthma; however, very few asthma medications have been evaluated in infants and young childr en. Budesonide inhalation suspension is an anti-inflammatory corticosteroid administered by nebulization that requires only passive inhalation and is therefore suitable for infants and young children who are unable to use cur rently available inhalation devices for the administration of asthma medica tions, The efficacy of budesonide inhalation suspension was evaluated in 3 US clinical trials. All 3 trials were randomized, controlled, double-blind, 12-week studies in children (6 month-8 years of age) with a primary diagno sis of chronic persistent asthma. Symptom assessments conducted in both the morning and evening were the primary efficacy variables. Although the chil dren were young, secondary efficacy evaluations included spirometry and pea k expiratory flow collected in both the morning and evening (in a subset of patients capable of performing these maneuvers). Patients who completed or were discontinued from the 12-week double-blind treatment phase were eligi ble to enter 52-week, open-label extension studies, The design of these 3 s tudies confirms the feasibility of the performance of placebo-controlled cl inical trials of asthma medications in pediatric patients and allows for ef ficacy evaluations based on symptom assessments together with pulmonary fun ction testing.