Clinical Pediatrics

To evaluate the safety and efficacy of a novel microbial lipase (NM BL) in a liquid formulation for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis, we performed a phase IIa proof-of-concept study. We conducted a doubleblind, randomized, placebo controlled crossover study in patients with cystic fibrosis (CF) and exocrine pancreatic insufficiency  (EPI). Adolescent and adult patients with CF were randomized to receive NM-BL or placebo for 1 week as replacement for their usual pancreatic enzyme formulation. They were subsequently crossed over to the alternate study treatment. The coefficient of fat absorption (CFA) was evaluated as primary end point. Symptoms and adverse events were evaluated as secondary end points. Thirty one patients were randomized in the study and 22 patients completed both treatment periods. During treatment with NMBL, the CFA was significantly higher (72.7%) compared to placebo (53.8%) with a difference between groups of 18.8% (P<0.001). Subjective assessment of stool fat and stool consistency also improved under treatment with NM-BL. Adverse events was mostly gastrointestinal in nature and consistent with the underlying disease. Currently available pancreatic enzyme products are limited because of the lack of liquid formulations and being largely porcine based. The novel microbial lipase NM-BL was safe and effective in this short term trial. The trial provided clinical proof-of-concept for this novel microbial lipase as a treatment for EPI in CF.