Clinical Trial Attitudes Among Individuals with Alpha-1 Antitrypsin Deficiency

Purpose This study examined characteristics of clinical trials that influence interest in participation among individuals with alpha-1 antitrypsin deficiency (AATD). Procedures A cross-sectional survey was completed by individuals with AATD. Thirty-four items described characteristics of clinical trials, which were rated from 1 (would not participate) to 5 (highly interested in participating). Logistic regression was used to compare participants with high interest in trials (defined as scores of 4 or 5 on >50% of responses) to all remaining participants. Results Data were provided by 1664 participants (91.6% with lung disease, 16.3% with liver disease, 14.9% with lung and liver disease). Nearly one-third (31.8%) indicated that they would not participate in a trial if there was a chance of getting a placebo. If the trial included three liver biopsies, 53.3% would not participate. More than two-thirds (69.8%) of participants who were using augmentation therapy would not participate in a trial that required twelve months off therapy. Individuals with two or more exacerbations in the prior year were more likely to have high interest in trials (OR=1.4, 95% CI=1.1-1.7, p=.009). In addition, individuals with a score of 10 or higher on the COPD Assessment Test were more likely to have high interest (OR=1.4, 95% CI=1.1-1.8, p=.010). Conclusions A sizeable percentage of participants indicated that they would not participate in clinical trials that include a placebo, involve multiple liver biopsies, or involve discontinuing augmentation therapy. Individuals who are more affected by AATD have more interest in trial participation than individuals who are less affected.