A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference
(2012) Journal of Cystic Fibrosis — Vol. 11, n° 3, p. 231-236 (2012)
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- 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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Leonard, A., Lebecque, P., Dingemanse, J., & Leal, T. (2012). A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference. Journal of Cystic Fibrosis, 11(3), 231-236. https://doi.org/10.1016/j.jcf.2011.12.004 (Original work published 2012)