The Approval was based on data from a randomized, double-blind, multicenter clinical trial of 374 patients with HIV who were on stable antiretroviral therapy and had a history of diarrhea lasting 1 month or longer. Diarrhea was defined as either persistently loose stools despite regular use of antidiarrheal medication or one or more watery bowel movements per day without regular use of medications. The median number of daily watery bowel movements was 2.5 per day. The trial was designed to measure clinical response, defined as the number of patients who had two or fewer watery bowel movements weekly.
Results showed that 17.6% of patients randomized to crofelemer 125 mg twice daily experienced clinical response, compared with 8% taking placebo (P < 0.01). In some patients, a persistent antidiarrheal effect was seen for 20 weeks. Upper respiratory tract infection, bronchitis, cough, flatulence, and increased levels of the liver enzyme bilirubin were common adverse events observed in the trial.