Outcomes and Statistical Analysis
Cancer
ADVERSE EVENT, PULMONARY SARCOIDOSIS, ADVERSE EVENT, DISEASE, SECONDARY, CANCER
The primary end point was to evaluate the safety and tolerability of efzofitimod vs placebo in patients with pulmonary sarcoidosis. Adverse events (AEs) were recorded from the date of informed consent and coded using the Medical Dictionary for Regulatory Activities version 24.0. TEAEs were defined as any AE or worsening of an existing condition after initiation of the study drug through 30 days after the last study. The intensity of each AE was rated by the masked investigator using the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Please refer to For the secondary outcome of potential corticosteroid-sparing effect of efzofitimod, the analysis included the time-adjusted area under the receiver operating characteristic curve from baseline to week 24 for each patient and a corresponding area under the receiver operating characteristic curve for the posttaper period (day 51 through end of study). The time-adjusted area under the receiver operating characteristic curve approximates the average daily corticosteroid dose per patient over the respective period. The development of antidrug antibody and Jo-1 antibodies (antibodies that recognize histidyl-tRNA synthetase) was used to summarize immunogenicity.Exploratory outcomes evaluated the change from baseline in lung function through week 24. The change in PRO scores from baseline to week 24 also were assessed as follows: SAT, sarcoidosis-specific patient-reported outcomes of impact of disease and response to therapyThe primary analysis population (safety set) comprised all patients who received any amount of study drug and was based on the actual treatment received. The primary efficacy analysis was the modified intention-to-treat population, defined as all randomized patients who received at least one administration of study drug.Statistical analyses were performed in an exploratory manner to reflect the phase 1/2 nature of the study. Continuous variables were summarized using descriptive statistics (number, mean ± SD, and median [interquartile range]). Categorical variables were summarized with the number and percentage of patients within each classification. Any calculated 