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
, David Schaeffer, MD 2, Richard C. Ahrens, MD 3, Natalie Sollo, MD 4, Steven Strausbaugh, MD 5, Gavin Graff, MD 6, Raksha Jain, MD 7, Stephan Witte, PhD 8, Kristin Forssmann, MD 8Abstract |
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 numéro
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