Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma

Patient disposition. ^∗A patient could be excluded for more than 1 reason. ^∗∗One patient subsequently received placebo but was not randomized to treatment; therefore, the patient was excluded from the intent-to-treat (ITT) population (n = 206) but included in the safety population (n = 207). Fifty-one patients were excluded from the modified ITT (mITT) population because of noncompliance with GCP study conduct issues (mITT population: omalizumab [n = 384], placebo [n = 192]). ^∗∗∗The sponsors conducted site audits as part of the study monitoring procedures. Concerns were raised regarding the manner in which the trials were conducted and data collected for 3 sites in particular. As a result of the audit findings at 2 of the sites, all randomized patients were discontinued from study drug and the sites were closed, and efficacy data were excluded. The third site with less significant issues was closed to further enrollment, but all randomized patients were allowed to continue until they completed the trial. Sites were instructed to record the reason for withdrawal as “admin problems” when special circumstances occurred, such as study closure or cancellation, or when patients relocated and could no longer participate.

Clinically significant asthma exacerbation rates over a period of 24 weeks (primary outcome; A) and 52 weeks (B) in patients with moderate-to-severe asthma treated with add-on omalizumab (n = 384) or placebo (n = 192) to an optimized asthma program (mITT population).