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Updated: Jan 8, 2026
Standardized Measurement of Nasal Membrane Transepithelial Potential Difference NPD
Published on: September 13, 2018
A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference
Anissa Leonard1, Patrick Lebecque, Jasper Dingemanse
1Pediatric Pulmonology & Cystic Fibrosis Unit, Cliniques St Luc, Université catholique de Louvain, 10 Avenue Hippocrate, 1200 Brussels, Belgium. anissa.leonard@uclouvain.be
Background:
Preclinical data suggest that miglustat could restore the function of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis cells.
Methods:
Single-center, randomized, double-blind, placebo-controlled, crossover Phase II study in 11 patients (mean±SD age, 26.3±7.7 years) homozygous for the F508del mutation received oral miglustat 200 mgt.i.d. or placebo for two 8-day cycles separated by a 14-day washout period. The primary endpoint was the change in total chloride secretion (TCS) assessed by nasal potential difference.
Results:
No statistically significant changes in TCS, sweat chloride values or FEV(1) were detected. Pharmacokinetic and safety were similar to those observed in patients with other diseases exposed to miglustat.
Conclusions:
There was no evidence of a treatment effect on any nasal potential difference variable. Further studies with miglustat need to adequately address criteria for assessment of nasal potential difference.
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