Safety and Efficacy of a Novel Microbial Lipase in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis: A Randomized Controlled Clinical Trial - 25/08/16
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Abstract |
Objective |
To evaluate the safety and efficacy of a novel microbial lipase (NM-BL) in a liquid formulation for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF) in a phase IIa proof-of-concept study.
Study design |
We conducted a double-blind, randomized, placebo controlled crossover study in patients with cystic fibrosis and exocrine pancreatic insufficiency. 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 was evaluated as the primary endpoint. Symptoms and adverse events were evaluated as secondary endpoints.
Results |
A total of 35 patients were randomized into the study and 22 patients completed both treatment periods. During treatment with NM-BL, the coefficient of fat absorption was significantly greater (72.7%) compared with placebo (53.8%) with a difference between groups of 18.8% (P < .001). Subjective assessment of stool fat and stool consistency also improved under treatment with NM-BL. Adverse events were mostly gastrointestinal in nature and were more common in the group receiving NM-BL.
Conclusions |
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. A larger phase 2 dose ranging trial is warranted.
Trial Registration |
ClinicalTrials.gov: NCT01710644.
Le texte complet de cet article est disponible en PDF.Keywords : clinical trial, cystic fibrosis, exocrine pancreatic insufficiency, pancreatic enzyme-replacement therapy
Abbreviations : BMI, CF, CFA, CNA, EPI, NM-BL, PERT, SAE, TEAE
Plan
All phases of this study were funded by Nordmark Arzneimittel GmbH & Co. KG, Uetersen Germany. K.F. is an employee of Nordmark. All other authors received investigator and/or consulting fees from Nordmark. The authors declare no conflicts of interest. |
Vol 176
P. 156 - septembre 2016 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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